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What to Know in Washington: Provisional Ballots May Play Role – Bloomberg Government

October 31st, 2020 5:53 pm

If the U.S. is still waiting to learn who the president will be days or even weeks after Tuesdays election, provisional ballots are likely to be at the center of any disputes.

They are the ballots cast by voters whose eligibility is questioned for some reason. Those ballots are set aside and held for a period of days after the election while workers determine whether they should be counted.

Experts say the number of provisional ballots this year may set a record, exceeding the 2.7 million cast in 2012 and almost 2.5 million cast in 2016, in part because some voters who requested ballots by mail are showing up at polls to vote in person. That could lead to late results in key battleground states such as Pennsylvania where President Donald Trump won by only 44,292 votes in 2016 if the race is close and the source of challenges and litigation.

Its just another one of those things that plays into the unusual nature of this election, G. Terry Madonna, director of the Center for Politics and Public Affairs at Franklin and Marshall College in Pennsylvania, said of provisional ballots.

Provisional ballots have come into play in the past. Democrat John Kerry held off conceding the 2004 presidential race to Republican George W. Bush until the morning after the election because he waited until it was clear the 157,714 provisional ballots cast in Ohio couldnt change the outcome.

In 2016, Trump carried Arizona by 91,234 votes, and there were 102,510 provisional ballots, according to the U.S. Election Assistance Commission. Pennsylvania had 26,451 provisionals, and that was before the commonwealth expanded voting by mail last year.

Greater use of mailed ballots, coupled with disruptions in mail service, may contribute to the bumper crop of provisional ballots. After initially urging voters to request mail-in ballots in response to the pandemic, Democratic leaders have been telling people to vote in person or deliver the ballot to an elections office or an approved drop box to avoid the risk of it being disqualified on technical grounds or having its delivery delayed by the U.S. Postal Service. Read more from Mark Niquette.

Photographer: David Paul Morris/Bloomberg

Voters who requested mail-in ballots but opt to vote in person could lead to more provisional ballots.

USPS Cites Covid, Holiday for Record Delays: The U.S. Postal Service delivered 80.85% of first-class mail on time for the week ending Oct. 23, compared with previous weeks figure of 85.58%, according to a news release. The agency listed factors including high mail volume, Covid-19 impacts, the Oct. 12 federal holiday and ongoing efforts to prioritize ballots for the Nov. 3 election. The on-time rate is the lowest of the year, according to the Save the Post Office website that tracks the agency. Read more from Todd Shields.

BGOVs Downballot Races to WatchReddit AMA: Join Bloomberg Governments elections guru Greg Giroux this afternoon on Reddit for an AMA on the congressional elections, where hell take questions on the races to watch. Follow his AMA here at 2 p.m. today.

Biden Win Would Offer Path to Kill Pipeline: Days after taking office, Trump cleared the way for construction of an oil pipeline in the Midwest that had been the focus of months of opposition by climate activists, celebrities and Native Americans. Now opponents of the Dakota Access Pipeline are pressuring Joe Biden to take the extraordinary step of returning the favor should he win the White House. Analysts said they couldnt recall a president shutting down an operating pipeline before, which is why its being viewed as a litmus test of how far hed go to appease environmentalists who have supported him. Read more from Ari Natter.

Biden Pledges Not to Extort South Korea: Biden pledged not to use the threat of cutting U.S. troop levels in South Korea as a bargaining chip, after the Trump administration demanded Seoul pay far more for American protection. Biden said if he wins the U.S. election next week, he wont be extorting Seoul with reckless threats to remove our troops, according to a special contribution he made to South Koreas Yonhap News Agency published today. Read more from Jon Herskovitz.

Trumps China Scorecard Shows Many Defeats: Trump ran for office pledging to rewrite the U.S.s economic ties with Beijing, which he blamed for hollowing out Americas manufacturing base and impoverishing its workers. But his four years in the White House have shown limited impact on the metrics he laid out. U.S. companies cite much the same concernsand the same growth objectiveswith regard to China today as they did when Trump took office. The unprecedented trade war that Trump launched, breaking GOP free-trade orthodoxy along the way, has ended up costing American factory jobs, not creating them, economists claim. The state support for Chinese enterprises that Trump pledged to confront remains intact.

Trumps term has, however, had a notable impact on American attitudes toward China. In time, that could prove the dynamic that affects economic ties in ways the current president has struggled to achieve. And it underscores that Washingtons policy on China is forever changed, regardless of who wins next Tuesdays election. Read more from Jenny Leonard.

Health-Care on the Ballot in Several States: Big money is betting on health-care initiatives in California, Oregon, and Colorado where voters are being asked Nov. 3 to add taxes on vaping, mandate that doctors always be on site when dialysis centers are open, and approve $5.5 billion in bonds for the sole stem cell research funding agency in the country. Tens of millions of dollars are fueling arguments over what to fund and why because, essentially, people are more interested in health-care initiatives, said Arturo Vargas Bustamante, associate professor in the Department of Health Policy and Management at the University of California, Los Angeles. Read more from Joyce E. Cutler.

Gerrymander Power on the Line: A shift toward independent redistricting commissions helped spur national political parties to spend record cash in the hope of controlling the next congressional redistricting. The outcomes in just a few races in a few states will determine the Republican Partys chances of winning congressional majorities for the next decade. And were fighting like hell to win, Republican State Leadership Committee President Austin Chambers said in an email. His group raised $23 million in the third quarter and is targeting 115 districts that could hold the keys to a 136-seat swing in the U.S. House.

Democrats are fighting money with money. The Democratic Legislative Campaign Committee has pledged to spend $50 million in state races this cyclemore than six times what it spent in 2010. Read more from Alex Ebert.

Biden Courts Latino Voters as Tie-Breakers: Biden appealed to Hispanic voters in Florida, a closely divided battleground state where a win next week would give him the presidency. The heart and soul of the country is at stake right here in Florida. Its up to you. You hold the key. If Florida goes blue, its over, Biden said in a neighborhood in Broward County, which stretches south from Fort Lauderdale toward Miami and is a key source of Democratic votes in the state. Read more from Jennifer Epstein.

Facebook Says Technical Hiccups Improperly Blocked Campaign Ads: Facebook revealed yesterday how internal technical glitches disrupted the delivery of some ads from the Biden and Trump campaigns, but said it made changes to resolve those hiccups in the run-up to the November election. The social media giants admission followed complaints from the Biden camp about how thousands of its ads were blocked. Facebook said in a blog post it spotted unanticipated issues affecting both campaigns, including technical flaws that caused a number of ads to be paused improperly. Read more from Sarah Frier.

Trumps Social Media Order Defeats Legal Challenge: The Trump administration defeated a challenge by Rock the Vote and Voto Latino to a recent executive order targeting social media companies that fact-check the presidents posts about the election, when a federal judge said yesterday that the get-out-the-vote groups lacked standing to bring the case. Judge William H. Orrick said the groups couldnt show the order regulated Facebook, Twitter, or another social media website in any way or credibly threatened them with prosecution. Read more from Mike Leonard.

BGOV OnPoint: Section 230 Internet Shield Faces Bipartisan Attacks.

Zeta Disrupts Early Voting in Georgia: Hurricane Zeta caused disruptions to early voting across the South yesterday, including in the key battleground states of Georgia and North Carolina. Zeta, now a tropical storm, knocked out power to more than 1 million homes and businesses as early in-person voting enters a final stretch with five days to go until Election Day. Friday is Georgias last day to vote while voters in North Carolina have until Saturday. Read more from Misyrlena Egkolfopoulou and Joe Ryan.

More Hawaiians Voted Early Than All of 2016: Early voting in Hawaii has already surpassed the total number of ballots cast in the state in the 2016 election. Data from the U.S. Elections Project, which tracks early voting, found Hawaii has seen turnout hit 104.5% of its total from the last election. More than half of all registered voters in Hawaii have already sent in their ballots, topping the record for the total number of votes cast in the state for a general election. Read more from Emma Kinery.

U.S. Sets Daily Virus Case Record: New U.S. cases topped 86,000, setting a new daily record, as the outbreak intensifies ahead of next weeks presidential election. Read more from Bloomberg News.

U.S. Buying Gowns from Untested Makers: Several U.S. companies that won almost half a billion dollars in government contracts to make hospital gowns appear to have too few workers and not enough factory space to complete the job when the awards were made, according to a Bloomberg Law analysis. At least 11 contractors and subcontractors began making protective gear only after the pandemic began, according to press releases and news reports. Of the 15 primary contractors, eight had never received a federal contract prior to 2020, according to federal government contracting records. The Defense Department says contractors went through a rigorous process and met stringent criteria.

Still, members of Congress are raising questions. Some of those contracts have been to companies that have very little experience with producing that kind of equipment, that the standards have not always been up to par, and its created some real challenges, Sen. Jeanne Shaheen (D-N.H.) said during a Senate Armed Services committee hearing Oct 1. Read more from Shira Stein.

Covid Relief Laws May Bolster ACA Legal Case: Republicans may have hurt their own legal case against Obamacare by repeatedly expanding on and amending the signature health law since 2017, most recently in their response to the coronavirus pandemic. You cant build on the ACAs protections for the Covid bills if the act doesnt exist, said Abbe Gluck, a professor of law and the founding faculty director of the Solomon Center for Health Law and Policy at Yale Law School.

The U.S. Supreme Court will assess legal arguments on the constitutionality of the Affordable Care Act on Nov. 10. What Congress intended to do with the ACA is a central line of dispute in the high stakes case, and the pandemic has given supporters of the law a new opportunity to prove it was meant to stay. Read more from Lydia Wheeler.

New Projection Shows Higher Death Toll: The Institute for Health Metrics and Evaluation, an influential modeling group, is projecting a higher death toll in the U.S. amid the surge in Covid cases and hospitalizations. The group is now projecting about 405,000 deaths due to the virus by Feb. 1, representing a nearly 20,000 increase from a prior projection of 386,000. Europe is seeing a surge right now and Europe is ahead about a month from the United States, said Ali Mokdad, a professor of health metrics sciences with IHME. So basically we are watching what would unfold here in the United States. Read more.

CDC Evictions Moratorium Upheld by Judge: A federal judge ruled against landlord groups trying to block the U.S. Centers for Disease Control and Preventions national moratorium on evictions amid the coronavirus crisis. U.S. District Judge J.P. Boulee in Atlanta yesterday rejected a motion for a preliminary injunction brought by the New Civil Liberties Alliance and National Apartment Association, which represents some 85,000 landlords responsible for 10 million rental units. The judge said the public interest in curtailing the spread of Covid outweighed the landlords economic interest. Read more from Chris Dolmetsch.

Trump Rule Leads Contractors to Cut Training: Trumps recent attempt to curb corporate diversity training will linger even if he loses Tuesdays election to Biden. Already, federal contractors are unsure of what they can say about race. More than 300 events, training programs, research projects and other diversity-related activities have been delayed or canceled because of concern about a Sept. 22 executive order aimed at banning federal contractors and agencies from using terms in diversity training that the administration considers divisive and illegal, according to the African American Policy Forum, a social justice think tank. A Labor Department official said last week that the agencys already received more than 100 complaints via a government hotline to report possible violations. Read more from Paige Smith and Jeff Green.

Hackers Plan More Attacks on U.S. Hospitals: A Russia-based ransomware group responsible for a new wave of attacks against U.S. hospitals is laying the groundwork to cripple at least ten more, according to the cybersecurity firm Prevailion. Prevailions analysis comes a day after the FBI and two other federal agencies issued a warning about an imminent and credible threat to hospitals and health-care providers from cyberattacks, including ransomware capable of locking entire computer networks. The hacking group responsible has already hit at least nine hospitals in three weeks, crippling critical computer systems and demanding multimillion-dollar ransoms. Read more from Alyza Sebenius, William Turton and Michael Riley.

Insurers Must Post Out-of-Pocket Medical Costs: Health insurers will have to post on the internet personalized out-of-pocket costs to patients under a new rule finalized yesterday. Insurance plans will also be required to make their in-network negotiated rates with doctors and allowable out-of-network rates available to the public, according to the multiagency rule. They will be required to post their negotiated pharmaceutical prices and the historical net prices for all their covered drugs. Read more from Shira Stein, Lydia Wheeler and John Tozzi.

How the Google Case Could Play Out: Google and the Justice Department are set to face each other in court today for the first time since the government sued the company for illegally monopolizing internet search. The hearing marks the first time attorneys for Google and the government will come together to start mapping out the the litigation process. It will set the stage for whats shaping up to be a drawn-out court battle that could ultimately weaken Googles gatekeeper position over the web if the government is successful. Vicky Graham lays out how to follow the case as it winds its way through federal court.

737 Jet Crash Victims Demand U.S. Records: The relatives of the 157 people who died in the crash of a Boeing 737 Max in Ethiopia are demanding more records from U.S. safety agencies, according to a pair of letters sent on the second anniversary of another accident involving the plane. A letter to the National Transportation Safety Board, which assisted in the investigation of both crashes, accused it of an unreasonable pro-secrecy stance. Alan Levin has more.

Gray Wolf Protections Stripped, Stirring Lawsuit Threats: The Interior Department announced yesterday it will strip endangered species protections from the gray wolf. Environmental groups vowed to challenge the decision in court, saying it violates the Endangered Species Act by ignoring the best available science. If Democrat Joe Biden wins the presidency, his administration could seek to overturn the move. Todays action reflects the Trump administrations continued commitment to species conservation based on the parameters of the law and the best scientific and commercial data available, said Interior Secretary David Bernhardt. Stephen Lee and Kellie Lunney have more.

Administration Appeals Court Order on Visa Ban: The Trump administration is appealing a California federal courts Oct. 1 ruling that blocked the departments of State and Homeland Security from barring entry to the U.S. for certain temporary work visa holders. In their filing, U.S. Justice Department lawyers signaled theyll ask the San Francisco-based U.S. Court of Appeals for the Ninth Circuit to reinstate Trumps proclamation in full, Genevieve Douglas reports.

To contact the reporters on this story: Zachary Sherwood in Washington at zsherwood@bgov.com; Brandon Lee in Washington at blee@bgov.com

To contact the editors responsible for this story: Giuseppe Macri at gmacri@bgov.com; Loren Duggan at lduggan@bgov.com; Michaela Ross at mross@bgov.com

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Impact of COVID-19 on Animal Cell Culture Market 2020 | Size, Growth, Demand, Opportunities & Forecast To 2026 | Life Technologies, Thermo Fisher,…

October 31st, 2020 5:53 pm

Animal Cell Culture Market research report is the new statistical data source added by A2Z Market Research.

Animal Cell Culture Market is growing at a High CAGR during the forecast period 2020-2026. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

Animal Cell Culture Market research is an intelligence report with meticulous efforts undertaken to study the right and valuable information. The data which has been looked upon is done considering both, the existing top players and the upcoming competitors. Business strategies of the key players and the new entering market industries are studied in detail. Well explained SWOT analysis, revenue share and contact information are shared in this report analysis.

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Top Key Players Profiled in this report are:

Life Technologies, Thermo Fisher, GE Healthcare, Corning (Cellgro), Takara, HiMedia, Atlanta Biologicals, PromoCell, Sigma-Aldrich, Zenbio, BD, Lonza, CellGenix, Merck Millipore

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Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Animal Cell Culture market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Animal Cell Culture markets trajectory between forecast periods.

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Global Animal Cell Culture Market Segmentation:

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Classical Media & SaltsSerum-free MediaStem Cell Media

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Biopharmaceutical ManufacturingTissue Culture & EngineeringGene TherapyCytogenetic

Regions Covered in the Global Animal Cell Culture Market Report 2020:The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

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Table of Contents

Global Animal Cell Culture Market Research Report 2020 2026

Chapter 1 Animal Cell Culture Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Animal Cell Culture Market Forecast

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Impact of COVID-19 on Animal Cell Culture Market 2020 | Size, Growth, Demand, Opportunities & Forecast To 2026 | Life Technologies, Thermo Fisher,...

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Lesaffre and Recombia Biosciences to advance innovative gene editing technology through a strategic partnership – PRNewswire

October 31st, 2020 5:52 pm

A strong commitment to innovationWith its partnership with Recombia, Lesaffre is investing in major pioneering technology. The ability to generate thousands of yeast strains in parallel, combined with laboratory automation, is expected to exponentially accelerate development of projects in the areas of health, the environment, and energy. The partnership also signifies Lesaffre's entry into the world of Synthetic Biology, considered to be the major biotechnological opportunity of this decade.

"This kind of partnership exemplifies an innovative way that industry can support and foster progress in Biotechnology. Through collaboration with scientists and entrepreneurs, we will be able to find new solutions, which will be beneficial for the future, especially in health or in environment protection," says Antoine Baule, Chief Executive Officer of Lesaffre.

An exclusive technologyRecombia Biosciences was founded by three Stanford University researchers in 2019 as a spin-off from the prestigious Stanford Genome Technology Center (SGTC).Recombia'stechnologies are based upon techniques that increase the efficiency of genome editing and enable engineering of yeast at very high throughput. The strategic collaboration with Lesaffre aims to advance Recombia's proprietary gene editing technologies to identify new yeast strains, discover novel yeast physiology of industrial relevance and optimize the production of biosourced ingredients and biofuels.

"We are excited to be working with Lesaffre on moving our gene editing programs forward," says Dr. Justin Smith, CEO of Recombia. "We see tremendous potential to leverage our expertise in genome editing and synthetic biology to develop new and innovative fermentation solutions and products."

Recombia is exclusively licensing four genome engineering technologies from Stanford University for their work.

"While precision genome editing has certainly advanced recently, there are still challenges, especially in making many genetic changes in parallel," said Dr.Bob St.Onge, COO and co-founder of Recombia Biosciences. "Recombia's technologies enable industrial yeast strain engineering by dramatically increasing the efficiency of high-throughput genome editing."

St.Onge and Smith co-founded the company with Professor Lars Steinmetz. The team has had a long working relationship at the SGTC.

"I am very excited to see the technologies we developed in academia applied in the industrial sector," said Steinmetz. "The Genome Technology Center has a long history of genomics technology development. I'm confident Recombia will continue in the tradition of the other successful companies that have spun out of the SGTC."

"The technology has broad utility and can be readily applied also to the development of non-genetically modified organisms,"says Carmen Arruda, Lesaffre R&I Manager. "With Recombia, Lesaffre can now explore a larger space of metabolic engineering hypotheses, develop prototype organisms at a faster pace, accelerate the design of appropriate selections and screenings of strains generated by classical breeding methods. We are excited to see what the future holds."

Working together to better nourish and protect the planetAs a global key player in the field of fermentation,Lesaffre is committed to continuing its investments in research and development to contribute to a safer, healthier and more natural world by developing the potential of micro-organisms, such as yeasts or beneficial bacteria.

More information about Recombia Biosciences at http://www.recombia.com

More information about Lesaffre at http://www.lesaffre.com

SOURCE Recombia Biosciences

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Integrity and Trust | Harvard Medical School – Harvard Medical School

October 31st, 2020 5:52 pm

Leaders from across academia, government and industry gathered to discuss regulatory science at the 2020 Global Conference on Regulatory Science. Top row, left to right: Peter Sorger, Amy Abernathy, George Daley, Norman Sharpless. Bottom row, left to right: Adam Palmer, Helga Gadarsdottir, Peter Mol, Steve Goodman.

Speakers and panelists from across academia, government and industry convened to discuss the future of the evaluation and regulation of new medicines at the first annual Global Conference on Regulatory Science, held virtually on Oct. 20 and 21.

While machine learning and data science were the conference themes, fundamental issues of integrity, transparency and patient trust were a refrain.

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The importance of these underlying issues has been starkly illuminated by the challenges posed by the COVID-19 pandemic, said Margaret Hamburg, former commissioner of the FDA and one of the events keynote speakers.

Even with all the best science, we can't generate the change we hope for if people don't trust it. Even a safe and effective vaccine won't help control the COVID-19 pandemic if people won't take it, said Hamburg.

For me, and I suspect or most of you, this is just enormously worrisome,"she added. "It's a powerful reminder that integrity and the trust it generates is such an essential foundation of everything else.

Scientific discoveries and technologies with the potential to transform human health emerge almost daily, and regulatory agencies like the FDA and its peers around the world have faced mounting challenges as they strive to keep up with the accelerating pace of innovationchallenges that have been amplified by the urgent need for therapeutics and vaccines for COVID-19.

Building trust among patients, balancing careful testing with timely approvals for potentially life-saving medicines, and other key topics were addressed over the course of the two-day conference, which was hosted by the Harvard-MIT Center for Regulatory Science (CRS), a partnership between Harvard University, MIT and the FDA that aims to further develop and improve the science of how drugs and other products are evaluated and brought to market.

Regulatory science is an increasingly compelling opportunity for fundamental innovation and real-world impact in creating safe and effective medicines, diagnostics and devices, said Peter Sorger, the Otto Krayer Professor of Systems Pharmacology at HMS.

Our goal is to try and improve these processes, make them more efficient, and critically, bring needed innovation to unmet medical needs, said Sorger, who co-directs the CRS with Florence Bourgeois, HMS associate professor of pediatrics at Boston Childrens Hospital, and Laura Maliszewski, executive director of the Harvard Program in Therapeutic Science and the Laboratory of Systems Pharmacology.

More than 650 attendees from around the world joined in the virtual discussions, which centered on the theme of how machine learning, data science and new technologiesincluding telemedicine and wearable devicesare changing drug development, clinical trials, medical care and more.

Speakers and panelists included Norman Sharpless, director of the National Cancer Institute; George Q. Daley, dean of HMS; Amy Abernathy, principle deputy commissioner of the FDA; and a broad range of leaders from academia, hospitals, government and industry.

The process of regulating new medicines and biotechnologies begins with scientists themselves, noted Daley.

Scientists bear the responsibility to participate in a shared governance model that invites transparent and independent oversight, he said, highlighting the Asilomar conference in 1975, when an international group of scientists came together to create voluntary guidelines for the manipulation of DNA, then a novel technology.

This established a precedent for self-regulation by scientists, which then informed subsequent regulation by government agencies.

The need for the scientific community to engage in self-governance has only increased in urgency, with the remarkably rapid emergence of CRISPR gene-editing approaches that can make permanent, heritable changes to an individuals DNA. At a meeting in 2015, Daley joined a cohort of scientists, including Jennifer Doudna, now a Nobel laureate, to strongly discourage germline genome editing.

We knew that this would have to be a prohibition that would be practiced by scientists and clinicians themselves, because the knowledge was emerging so rapidly it wasn't clear that the regulators were going to be ready to catch up, said Daley.

But in 2018, a rogue scientist illicitly edited the genomes of two embryos that were carried to term in China, sparking international controversy. If there is ever to be a possible safe and ethical path forward for emerging technologies such as germline editing, it must be paved by the open cooperation and collaboration of scientists, regulators, and importantly, the public, Daley said.

The payoff for this kind of cooperation can be enormous, and perhaps the best examples can be found in recent successes in the development and approval of new cancer medicines, said Sharpless.

I predict that 2020 will be the best year thus far for cancer drug approvals, said Sharpless. That progress has occurred during a time when the FDA has been besieged by a global pandemic.

A historic surge of new cancer medicines has entered the U.S. market in recent years, Sharpless added, a windfall that stems from decades of productive research on cancer biology and therapeutics.

An improved scientific understanding of cancer has led to the development of new medicines that have prompted new approaches to regulation by the FDA. Some cancer drugs demonstrate such efficacy in small-scale clinical trials, he noted, that it can become essentially unethical to withhold them while waiting for large phase III trials to finish.

This has been a change for the regulatory thinking of the FDA, and I would argue has been a change for the good of the patients, Sharpless said. It has made agents available to patients at a sooner date and led the pharmaceutical industry to develop cancer drugs knowing that they can get approval at an earlier stage.

The recent successes of cancer drugs are to be celebrated, but the question of how to replicate these successes in other diseases, such as neurodegeneration and other intractable diseases, remains a pressing concern, he said.

This question was discussed by conference speakers and panelists in many different contexts, particularly the potential of emerging technologies to reshape how clinical trials are conducted in the future.

A wealth of new technologies, from telemedicine to wearable devices, are allowing physicians and scientists to engage with patients in unprecedented ways. This could have a transformative impact in medicine in many ways, including by augmenting clinical trials, speakers said.

Such technologies could enable more frequent physician-patient interaction and the continuous monitoring of real-world data and evidenceproviding far more information than the intermittent site visits that most current trials use to collect data.

In addition, new technologies could help reduce disparities in access to clinical trials, panelists said, and allow for vastly improved patient recruitment, which would help ensure that new medicines are being evaluated on patients who have the best chance of benefiting.

If this potential is to be realized, patients must have confidence that their privacy and data are protected, said conference speakers and panelists.

In many ways, trust in data security and privacy are as important as any innovations in technology itself, panelists noted. This is a key issue for new digital medicine technologies and approaches, they added, and thoughtful and transparent regulation are critical.

Conference speakers also addressed a wide and diverse range of other issues, including how new technologies, such as artificial intelligence and digital pathology, are transforming clinical care and how large data sources like electronic medical records are linked and mined for insights into improving health.

The rapid growth of these and many other new technologies in health care present myriad complex issues for those tasked with evaluation and regulation, speakers and panelists said. And in many cases, as with genetic engineering, decision-making will require societal discourse.

As such, neutral forums to consider and debate new innovations, policies and regulationsone of the key functions of the CRS and its annual conferenceplay an increasingly important role in moving the complex discipline of regulatory science forward.

Central to this process is the ability to effectively collaborate around stakeholders, across academia, industry and regulatory agencies, said Bourgeois.

This is where the center comes in, serving as a platform to foster interdisciplinary and multi-stakeholder conversation, she said.

The remarkable discoveries and the acceleration and advances we are seeing in our understanding of diseases and how to treat themthese will most benefit patients if we have an efficient, rigorous and adaptable approach to the evaluation of the many rapidly emerging biotechnologies, Bourgeois said.

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Gene-edited crops and animals: Best-kept secrets in the fight against climate change – Genetic Literacy Project

October 31st, 2020 5:52 pm

Advances in gene editing over the past decade have given scientists new tools to tailor the biochemistry of nearly any living thing with great precision. Because the biosphereincluding trees, crops, livestock, and every other organismsis a major source and sink for greenhouse gases (GHGs), these tools have profound implications for climate change. Gene editing is unlocking new ways to enhance natural and agricultural carbon sinks, limit emissions from agriculture and other major GHG-emitting sectors, and improve biofuels. Congress should act now to open this new frontier for climate innovation.

Gene editing uses enzymesCRISPR Cas9 is the most well-knownto identify, remove, and replace segments of an organisms DNA, much like using a word processor to edit a document. These tools originated as defense mechanisms so that bacteria could remove foreign DNA inserted by predatory viruses. Researchers have adapted this cellular machinery to introduce beneficial traits into plants and animals. The techniques are new, but they build on nearly a half century of experience with conventional genetic engineering and hundreds of millions of years of evolution.

Zooming out from the microscopic level, gene editing offers novel solutions to a diverse set of emissions-related problems.

The Trillion Trees initiative recognizes plants unique ability: using photosynthesis to capture carbon. Yet the process is surprisingly inefficient.Scientistshave moved swiftly to use their new toolkit to try to improve it, and several breakthroughs have already been reported. Further progress might enableproductivity gainsof 50 percent in major crops, slashing emissions radically, raising output per acre, and bolstering farmers incomes.

The decomposition and transport of wasted food accounts for the single largest portion of agricultural GHG emissions. Companies are already selling gene-editedsoybean oilwith a longer shelf life andpotatoesthat resist bruising, both of which reduce waste.

Next-generation biofuels from switchgrass, which grows easily on otherwise non-arable land, could power sustainable, low-carbon transport. The hitch has been that this plants key ingredient, cellulose, is hard to break down. Gene editing may open up this abundant resource by optimizing microbes that can efficiently process cellulose, yielding low-cost biofuels and spurring rural development.

The worlds 1.4 billion cattle account for about6 percentof global agriculture GHG emissions, in large part because of methane in their burps. Some cattle emit far less methane than others because of specific microbial populations in their digestive tracts. Gene editing could allow this trait to spread across herds,reducing emissions.

Gene editings enormous promise for solving societal problems, including climate change, has been slowed by concerns that it is neither natural nor safe. These concerns are misplaced. Humans have used breeding to shape the genomes of crops and livestock since the dawn of agriculture. Our new gene editing toolkit has been used by nature for hundreds of millions of years. Most important, in eleven major studies over the past four decades, the U.S. National Academy of Sciences hasfoundno new hazards in gene edited or genetically engineered products. Other authoritative bodies around the world have drawn the same conclusion, which has been confirmed by vast experience.

The urgency of the climate challenge is becoming clearer with each passing season as severe storms, droughts, fires, and other disasters become more frequent at home and around the world. Congress should take action today to accelerate gene-edited climate solutions. First, legislators should eliminate regulatory burdens that disincentivize innovation in gene-edited technologies and contribute little to human or environmental safety. Current regulations on gene-edited products have addedtens of millions of dollarsand multiple years to their development without delivering commensurate benefits for health, safety, or the environment.

Second, Congress should create a new agency to support agricultural research into high-reward biological technologies including gene editing. The ARPA-Terra Act of 2019 (S.2732) introduced by Sen. Michael Bennet would do so, emulating the highly successful models of the Defense Advanced Research Projects Agency (DARPA) and the Advanced Research Projects Agency-Energy (ARPA-E).

Finally, Congress should encourage innovative farmers to adopt new gene-edited crops and livestock to demonstrate their value and speed wider deployment. Existing tax credits for carbon capture could be expanded as these nascent products come to market.

Although gene editing is less than a decade old, it is already abundantly clear that it will be a powerful tool to address climate change. The science is ready and waiting for Congressional action.

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Gene-edited crops and animals: Best-kept secrets in the fight against climate change - Genetic Literacy Project

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ETFs in Focus on Bayer’s Bet on Gene Therapy – Yahoo Finance

October 31st, 2020 5:52 pm

Merger and acquisition activities have heated up in recent months and the niche spaces are in the limelight. After the telemedicine industry, gene therapy stocks jumped on the bandwagon. Gene therapy is a technique that uses genes to treat or prevent disease.

German drugmaker Bayer has made a big bet on gene therapy by announcing the acquisition of U.S. biotech firm Asklepios BioPharmaceutical for as much as $4 billion. The proposed acquisition will provide Bayer access to the adeno-associated virus (AAV) gene therapy platform and a pipeline led by clinical-phase treatments for Parkinsons, Pompe disease and congestive heart failure. Notably, AAV therapies offer improved efficacy, immune response, and tissue and organ specificity.

Additionally, the transaction will complements Bayers 2019 acquisition of BlueRock Therapeutics and advances its efforts to create platforms with the potential to have an impact on multiple therapeutic areas (read: Genomics ETFs Surge on Nobel Prize for Gene-Editing Pioneers).

Under the terms of the deal, Bayer will pay an upfront consideration of $2 billion and potential success-based milestone payments of up to $ billion. About 75 % of the potential milestone-based contingent payments are expected to be due during the course of the next five years and the remaining amount thereafter.

The deal, pending regulatory approvals, is expected to close during the fourth quarter of 2020. Once the deal closes, Bayer will allow Asklepios, known as AskBio, to operate autonomously as part of a new cell and gene therapy unit in a bid to preserve its entrepreneurial culture. The cell and gene therapy unit will bundle Bayer's activities in this area moving forward in order to establish an innovation ecosystem for the participating partners, the German company said (see: all the Healthcare ETFs here).

The proposed deal will provide a boost to the gene therapy industry. Below, we have highlighted four ETFs that are expected to benefit from Bayers entrance into the gene therapy space:

ARK Genomic Revolution Multi-Sector ETF ARKG

This actively managed ETF is focused on companies that are likely to benefit from extending and enhancing the quality of human and other life by incorporating technological and scientific developments, and advancements in genomics into their business. With AUM of $2.9 billion, the fund holds 47 stocks in its basket and has 0.75% in expense ratio. It trades in an average daily volume of 978,000 shares (read: 4 Sector ETFs That Have Doubled This Year).

Invesco Dynamic Biotechnology & Genome ETF PBE

This fund follows the Dynamic Biotech & Genome Intellidex Index and provides exposure to companies engaged in the research, development, manufacture and marketing and distribution of various biotechnological products, services and processes and companies that benefit significantly from scientific and technological advances in biotechnology and genetic engineering and research. It holds 31 stocks in its basket and charges 57 bps in annual fees. The ETF has managed $229.9 million in its asset base while trades in a light volume of 6,000 shares per day. Expense ratio comes in at 0.57%. The product has a Zacks ETF Rank #3 (Hold) with a High risk outlook.

Global X Genomics & Biotechnology ETF GNOM

This product seeks to invest in companies that potentially stand to benefit from further advances in the field of genomic science, such as companies involved in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics and biotechnology. It follows the Solactive Genomics Index, holding 40 stocks in its basket. This ETF has accumulated $68 million in its asset base and charges 50 bps in annual fees. It trades in average daily volume of 31,000 shares (read: Why You Should Invest in Genomics ETFs).

iShares Genomics Immunology and Healthcare ETF IDNA

This ETF provides access to companies at the forefront of genomics and immunology innovation by tracking the NYSE FactSet Global Genomics and Immuno Biopharma Index. Holding 46 stocks in its basket, the fund has gathered $166.2 million in AUM and trades in moderate average daily volume of 58,000 shares. It charges 47 bps in annual fees.

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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportInvesco Dynamic Biotechnology Genome ETF (PBE): ETF Research ReportsARK Genomic Revolution ETF (ARKG): ETF Research ReportsGlobal X Genomics Biotechnology ETF (GNOM): ETF Research ReportsiShares Genomics Immunology and Healthcare ETF (IDNA): ETF Research ReportsTo read this article on Zacks.com click here.Zacks Investment ResearchWant the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

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Sugar beet industry flips narrative on GMO crops – Idaho State Journal

October 31st, 2020 5:52 pm

It's only been a few years since U.S. sugar beet farmers faced a potential financial crisis due to negative public perceptions about food products derived from biotechnology.

Nowadays, however, the sugar beet industry is flipping the narrative, capitalizing on what was once its Achilles heel its universal adoption of GMO seed.

Since, 2009, the nation's sugar beet crop has been almost entirely planted in seed genetically modified to resist glyphosate herbicide, which is produced at Bayer's Soda Springs plant. Lately, to strike a chord with an increasingly environmentally conscious consumer base, the sugar beet industry has been touting how biotechnology has made its crop production system far more sustainable.

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Idaho is the No. 2 state in production of sugar beets by ton.

"We have lots of data," said Scott Herndon, vice president and general counsel with the American Sugarbeet Growers Association. "We submitted something to the National Academy of Sciences where we identified 25 environmental gains achieved through biotech seeds related to water, soil and air."

Herndon explained the gene added to confer glyphosate resistance to GMO beets is entirely removed in the processing of sugar. He referenced laboratory testing conducted by his industry proving finished sugar raised by American sugar beet farmers is identical to organic sugar and cane sugar in general.

Yet efforts to pass GMO labeling laws prior to 2016 in states including Vermont, Washington State, Oregon and California resulted in conventional cane sugar enjoying a whopping price advantage of roughly 7 cents over beet sugar, said Luther Markwart, executive vice president of American Sugarbeet Growers Association.

"That's huge," Markwart said.

Furthermore, the large Pennsylvania-based candy manufacturer Hershey Co. removed GMO beet sugar from many of its products in 2015 in favor of conventional cane sugar. In explaining the decision, Hershey affirmed GMO ingredients are safe but emphasized its commitment to openness and transparency.

"People care about the food they eat," Hershey posted on its website. "They want to know what's inside and they want to have choices so that snack options meet their expectations."

Rather than providing the public with transparency, Markwart believes mandatory labeling of beet sugar would be misleading, causing people to falsely believe beet sugar and cane sugar are somehow different.

Under the Obama Congress passed a preemptive law in 2016 exempting beet-derived sugar from state GMO labeling requirements. It took effect in December of 2018, though food companies are still allowed to voluntarily state that a product is derived from a bioengineered crop.

"Once you got the federal preemption, all of a sudden the beet and cane price came back together," Herndon said.

Labeling proponents, however, note that surveys consistently find consumers support GMO labeling by wide margins, believing people who are skeptical of a technology that is found in more than three-quarters of the processed foods in supermarkets should have the ability to avoid it.

"While many in the scientific community assert that GMO foods are not toxic and are safe, a significant number of scientists are sounding the alarm," Mark Fergusson, CEO of Down to Earth Organic & Natural, said in an essay posted on his organization's website. "They say genetic engineering poses risks that scientists simply do not know enough to identify."

Fergusson encouraged the public to choose foods with the organic seal, certifying that GMOs were not used in production.

GMO advocates argue that reputable scientific data evidencing health risks of GMO technology has yet to be produced.

The industry's comments to the National Academy of Sciences on Sept. 9, 2015, for example, cited several studies on the safety of GMO crops, including a 2011 summary report by the European Commission covering a decade of publicly funded research, 130 research projects and 500 research groups concluding "there is no scientific evidence of higher risks of GE crops for food and feed safety, or to the environment."

The environmental benefits of GMO crops, however, are well documented, Markwart said.

When beets were raised conventionally, Markwart said herbicides applied about four times per year to control weeds stymied crop development, essentially taking a month of growth off of the final yield. Glyphosate applications in GMO beets don't set development back whatsoever, enabling farmers to produce more with fewer farming inputs.

Furthermore, GMO beets don't require hand weeding and mechanical cultivation between rows, saving farmers on labor costs and avoiding soil disturbance, which dries out soil and releases greenhouse gases.

The sugar beet industry points to a 2002 study by the National Center for Food and Agriculture Policy evaluating eight of the most commonly used herbicides, finding glyphosate posed the least potential risk. Furthermore, glyphosate binds tightly to the soil and is less likely to contaminate groundwater, according to the industry's literature.

According to USDA data, sugar beet yields increased by 19% with glyphosate-resistant seed from 2008 through 2015 compared with the conventional average. The percentage of sugar in each beet also increased dramatically.

Brad Griff, executive director of the Idaho Sugarbeet Growers Association, said the Gem State's farmers have increased their yields by roughly 10 tons per acre on average since the implementation of GMO sugar beets.

"Before the GE sugar beet we never had reached 18% sugar. Now for three of the last six years we've been at or above 18% sugar," Griff added. "That's all been accomplished while reducing pesticide use by 85% and reducing fuel use by 60%."

In its Agriculture Innovation Agenda, USDA asked farmers of various commodities to plan strategies to increase their output by 40% while cutting their environmental impacts in half. The sugar beet industry submitted comments toward that effort focusing largely on achieving the goals through improved genetics.

Looking ahead, Markwart anticipates sugar beet seed engineered with multiple-trait tolerance to three herbicides glyphosate, dicamba and glufosinate should be released by 2026. He explained the new seed should help farmers avoid the onset of glyphosate-resistant weeds by enabling them to use multiple modes of action.

The industry also sees great promise in gene editing, which allows crop breeders to silence or amplify existing traits in a plant's genetics rather than introducing desirable foreign traits.

Herndon believes sustainability should be viewed as a "three-legged stool" factoring in social, economic and environmental costs. He said farmers are the lifeblood of rural economies and have a social contract to help keep their rural communities afloat. While it's important that they continually push the envelope to reduce their impacts on the environment, Herndon said farmers must also eke out a profit.

Markwart added, "We used to have hand labor. No one is going to go back and do that again. We have made major advances in efficiencies with this technology."

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Lesaffre and Recombia Biosciences apply gene editing technology in fermented ingredients production – FoodIngredientsFirst

October 31st, 2020 5:52 pm

30 Oct 2020 --- French yeast manufacturer Lesaffre and Recombia Biosciences have partnered to advance a gene editing technology for the sustainable production of fermented ingredients.

Using Recombias proprietary gene editing technologies, the partnership aims to generate thousands of new yeast strains while optimizing the production of biosourced ingredients.

We see tremendous potential to leverage our expertise in genome editing and synthetic biology to develop new and innovative fermentation solutions and products, says Dr. Justin Smith, CEO of Recombia Biosciences.

Click to EnlargeThe partnership signals Lesaffres entry into the world of Synthetic Biology, which the company notes is considered to be a major biotechnological opportunity of this decade.Recombia Biosciences was founded by three Stanford University researchers in 2019 as a spin-off from the Stanford Genome Technology Center (SGTC), in the US.

Its technologies are based upon techniques that increase the efficiency of genome editing and enable engineering of yeast at very high throughput.

The technology has broad utility and can be readily applied also to the development of non-genetically modified organisms, adds Carmen Arruda, R&I manager at Lesaffre.

With Recombia, Lesaffre can now explore a larger space of metabolic engineering hypotheses, develop prototype organisms at a faster pace, accelerate the design of appropriate selections and screenings of strains generated by classical breeding methods. Were excited to see what the future holds.

Entry into synthetic biologyThe partnership signals Lesaffres entry into the world of Synthetic Biology, which the company notes is considered to be a major biotechnological opportunity of this decade.

This kind of partnership exemplifies an innovative way that industry can support and foster progress in Biotechnology, says Antoine Baule, CEO of Lesaffre.

Through collaborations with scientists and entrepreneurs, we will be able to find new solutions, which will be beneficial for the future, especially in health or in environment protection.

Recombia is exclusively licensing four genome engineering technologies from Stanford University for their work.

Bridging industry with academiaWhile precision genome editing has certainly advanced recently, there are still challenges, especially in making many genetic changes in parallel, notes Dr. Bob St.Onge, COO and co-founder of Recombia Biosciences.

Recombias technologies enable industrial yeast strain engineering by dramatically increasing the efficiency of high-throughput genome editing, he remarks.

Click to EnlargeWhile precision genome editing has certainly advanced recently, there are still challenges ahead.

St.Onge and Smith co-founded the company with Professor Lars Steinmetz. The team has had a significant working relationship at the Stanford Genome Technology Center (SGTC).

Im very excited to see the technologies we developed in academia applied in the industrial sector, comments Steinmetz.

The Genome Technology Center has a long history of genomics technology development. Im confident Recombia will continue in the tradition of the other successful companies that have spun out of the SGTC.

Unlocking the genome for new ingredientsGenomic research is widely applicable across food-tech applications. For instance, this type of analysis is employed to map the chemical fingerprint of chocolate, using the genes of the tree that cacao pods are harvested from.

Also in this space, seed breeding specialist Equinom is leveraging its advanced breeding techniques to promote agricultural biodiversity. The company upholds its pivotal position in inducing better crop resilience and increased yield.

Meanwhile, a genomic study comparing historic and modern wheat varieties recently revealed an increase in dietary fiber and a decrease in acrylamide, indicating that white bread is not as unhealthy as it has often been portrayed.

Edited by Benjamin Ferrer

To contact our editorial team please email us at editorial@cnsmedia.com

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Polymerase Chain Reaction (PCR) Market expanding at a considerable CAGR from 2018 to 2026 – The Think Curiouser

October 31st, 2020 5:52 pm

Transparency Market Research (TMR)has published a new report titled,Polymerase Chain Reaction (PCR) Market: Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026.According to the report, theglobal PCR marketis projected to reach over US$ 7.0 Bn by 2026 expanding at a considerable CAGR from 2018 to 2026. North America is expected to dominate the global PCR market during the forecast period, due to high adoption of technologically advanced molecular diagnostic products, growing incidence of infectious diseases, rising prevalence of various types of cancer, growing trend of self-diagnosis of diseases, and affordability of high-cost testing processes for people in the region.

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Reagents Segment to Dominate the Global PCR Market

The report offers detailed segmentation of the global PCR market based on product and end-user. In terms of product, the market has been segmented into instruments, reagents, and consumables. The reagents segment held the leading market share in the year 2017 and is expected to expand at the highest CAGR during the forecast period. This is attributable to high consumption of reagents for the PCR technique. Introduction of advanced reagents specific to the type of test is expected to boost the global demand for PCR reagents in the near future. PCR reagents include template DNA, PCR primers and probes, dNTPs, PCR buffers, enzymes, and master mixes. PCR consumables mostly include PCR tubes, plates, and other accessories required to conduct PCR reactions. The instruments segment has been sub-categorized into standard PCR systems, RT PCR systems, and digital PCR systems. Among these, the digital PCR systems sub-segment is expected to witness growth at a significantly rapid pace during the forecast period. This is attributable to advantages of digital PCR systems such as precision, sensitivity, accuracy, reproducibility, direct quantification and multiplexing, and speed of the analysis.

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Pharmaceutical & Biotechnology Industry Segment Held a Major Share of the Global PCR Market in 2017

The PCR technique has been found to be useful in pharmaceutical and biotechnology research activities as well as microbial quality testing. The technique is also applied in genetic engineering. Genetic engineering is the key driver for the global PCR market. It is used to identify genes related to certain phenotypes includinggenetic disorders. Regular testing of the microbial load of raw materials and finished products is an important process in the pharmaceutical & biotechnology industry. Sophisticated analytical methods such as polymerase chain reaction (PCR) have been widely applied for quality control analysis in the pharmaceutical sector.

Market in Asia Pacific to Expand at a High CAGR

Molecular diagnosis has revolutionized the modern diagnosis technology. PCR has become a method of choice in early and accurate detection of diseases. Expansion by leading manufacturers of PCR products in the Asia Pacific region by strengthening of the distribution network and new product launches in developing countries of Asia Pacific are key factors likely to drive the PCR market in the region during the forecast period. Moreover, rise in the incidence of cancer and infectious diseases has resulted in increase in the demand for use of the PCR technique in clinical diagnosis of these diseases in Asia Pacific. For instance, according to the Korea Central Cancer Registry published in 2016, there were 217,057 cancer cases in South Korea in 2014. Moreover, in 2016, the WHO estimated that the Asia Pacific region has the second-highest number (i.e. 5.1 million) of people living with HIV across the world. Thus, Asia Pacific is expected to be the most lucrative market for PCR by 2026.

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Competition Landscape

Major players operating in the global PCR market are Bio-Rad Laboratories, Inc., QIAGEN N.V., F. Hoffmann-La Roche AG, Thermo Fisher Scientific, Inc. Becton, Dickinson and Company, Abbott, Siemens Healthcare GmbH (Siemens AG), bioMrieux SA, Danaher Corporation, and Agilent Technologies. Key players are expanding their product portfolio through mergers and acquisitions and partnerships and collaborations with leading pharmaceutical and biotechnology companies and by offering technologically advanced products.

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Transparency Market Research is a global market intelligence company providing global business information reports and services. Our exclusive blend of quantitative forecasting and trends analysis provides forward-looking insight for several decision makers. Our experienced team of analysts, researchers, and consultants use proprietary data sources and various tools and techniques to gather and analyze information.

Our data repository is continuously updated and revised by a team of research experts so that it always reflects latest trends and information. With a broad research and analysis capability, Transparency Market Research employs rigorous primary and secondary research techniques in developing distinctive data sets and research material for business reports.

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Aridis Pharmaceuticals to Present at the ROTH Capital Partners 2020 MedTech Innovation Forum on a COVID-19 Panel – BioSpace

October 31st, 2020 5:52 pm

SAN JOSE, Calif., Oct. 27, 2020 /PRNewswire/ -- Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, today announced the Company will present at the ROTH Capital Partners 2020 MedTech Innovation Forum on Wednesday, October 28, 2020. Dr. Hasan Jafri, Chief Medical Officer of Aridis Pharmaceuticals, will be a speaker on a panel entitled "Direct Antivirals and Other Agents Against SARS-CoV2 Virus."

Panel: Direct Antivirals and Other Agents Against SARS-CoV2 VirusDate: Wednesday, October 28, 2020Time: 10:30AM-11:50AM ET

Dr. Jafri will present a summary of the Company's recently published preclinical data of its COVID-19 inhaled mAb (AR-711). He will address the preclinical performance of AR-711, the advantages of direct lung delivery using nebulized aerosols, and the COVID-19 clinical program.

About AR-711

AR-711 is a fully human immunoglobulin 1, or IgG1, monoclonal antibody discovered from screening the antibody secreting B-cells of convalescent COVID-19 patients. AR-711 exhibits high affinity for SARS-CoV-2 spike protein, approximately 10-fold or higher than mAb candidates currently in late stage clinical testing. AR-711 was previously shown to be effective in prophylactic as well as therapeutic treatment modes in a SARS-CoV-2 viral challenge study. AR-711 is currently being developed as an inhaled, self-administered treatment for non-hospitalized patients suffering from mild to moderate COVID-19. AR-711 is also one the two mAbs in the company's AR-701 mAb cocktail, which is a separate program being developed as an intravenous treatment of moderate to severe, hospitalized COVID-19 patients.

About Aridis Pharmaceuticals, Inc.

Aridis Pharmaceuticals, Inc. discovers and develops anti-infectives to be used as add-on treatments to standard-of-care antibiotics. The Company is utilizing its proprietary PEXTM and MabIgX technology platforms to rapidly identify rare, potent antibody-producing B-cells from patients who have successfully overcome an infection, and to rapidly manufacture monoclonal antibody (mAbs) for therapeutic treatment of critical infections. These mAbs are already of human origin and functionally optimized for high potency by the donor's immune system; hence, they technically do not require genetic engineering or further optimization to achieve full functionality.

The Company has generated multiple clinical stage mAbs targeting bacteria that cause life-threatening infections such as ventilator associated pneumonia (VAP) and hospital acquired pneumonia (HAP), in addition to preclinical stage antiviral mAbs. The use of mAbs as anti-infective treatments represents an innovative therapeutic approach that harnesses the human immune system to fight infections and is designed to overcome the deficiencies associated with the current standard of care which is broad spectrum antibiotics. Such deficiencies include, but are not limited to, increasing drug resistance, short duration of efficacy, disruption of the normal flora of the human microbiome and lack of differentiation among current treatments. The mAb portfolio is complemented by a non-antibiotic novel mechanism small molecule anti-infective candidate being developed to treat lung infections in cystic fibrosis patients. The Company's pipeline is highlighted below:

Aridis' Pipeline

AR-301 (VAP). AR-301 is a fully human IgG1 mAb currently in Phase 3 clinical development targeting gram-positive Staphylococcus aureus (S. aureus) alpha-toxin in VAP patients.

AR-101 (HAP). AR-101 is a fully human immunoglobulin M, or IgM, mAb in Phase 2 clinical development targeting Pseudomonas aeruginosa (P. aeruginosa) liposaccharides serotype O11, which accounts for approximately 22% of all P. aeruginosa hospital acquired pneumonia cases worldwide.

AR-501 (cystic fibrosis). AR-501 is an inhaled formulation of gallium citrate with broad-spectrum anti-infective activity being developed to treat chronic lung infections in cystic fibrosis patients. This program is currently in a Phase 1/2a clinical study in healthy volunteers and CF patients.

AR-401 (blood stream infections). AR-401 is a fully human mAb preclinical program aimed at treating infections caused by gram-negative Acinetobacter baumannii.

AR-701 (COVID-19). AR-701 is a cocktail of fully human mAbs discovered from convalescent COVID-19 patients that are directed at multiple envelope proteins of the SARS-CoV-2 virus.

AR-711 (COVID-19). AR-711 is an in-licensed mAb that is directed against the receptor binding domain of the SARS-Cov 2 virus. The agent has the potential to be delivered both intravenously and by inhalation using a nebulizer.

AR-201 (RSV infection). AR-201 is a fully human IgG1 mAb out-licensed preclinical program aimed at neutralizing diverse clinical isolates of respiratory syncytial virus (RSV).

For additional information on Aridis Pharmaceuticals, please visit https://aridispharma.com/.

Forward-Looking Statements

Certain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. These statements may be identified by the use of words such as "anticipate," "believe," "forecast," "estimated" and "intend" or other similar terms or expressions that concern Aridis' expectations, strategy, plans or intentions. These forward-looking statements are based on Aridis' current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the need for additional financing, the timing of regulatory submissions, Aridis' ability to obtain and maintain regulatory approval of its existing product candidates and any other product candidates it may develop, approvals for clinical trials may be delayed or withheld by regulatory agencies, risks relating to the timing and costs of clinical trials, risks associated with obtaining funding from third parties, management and employee operations and execution risks, loss of key personnel, competition, risks related to market acceptance of products, intellectual property risks, risks related to business interruptions, including the outbreak of COVID-19 coronavirus, which could seriously harm our financial condition and increase our costs and expenses, risks associated with the uncertainty of future financial results, Aridis' ability to attract collaborators and partners and risks associated with Aridis' reliance on third party organizations. While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Aridis' 10-K for the year ended December 31, 2019 and Aridis' other filings made with the Securities and Exchange Commission. Forward-looking statements included herein are made as of the date hereof, and Aridis does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.

Contact:

Investor RelationsJason WongBlueprint Life Science Groupjwong@bplifescience.com(415) 375-3340 Ext. 4

SOURCE Aridis Pharmaceuticals, Inc.

View original content to download multimedia:http://www.prnewswire.com/news-releases/aridis-pharmaceuticals-to-present-at-the-roth-capital-partners-2020-medtech-innovation-forum-on-a-covid-19-panel-301160211.html

SOURCE Aridis Pharmaceuticals, Inc.

Company Codes: NASDAQ-NMS:ARDS

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Aviceda Therapeutics Announces Formation of Scientific Advisory Board – BioSpace

October 31st, 2020 5:52 pm

Oct. 27, 2020 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Aviceda Therapeutics, a late-stage, pre-clinical biotech company focused on developing the next generation of immuno-modulators by harnessing the power of glycobiology to manipulate the innate immune system and chronic, non-resolving inflammation, is announcing the members of its Scientific Advisory Board who will help shape ongoing development efforts.

The Aviceda Scientific Advisory Board includes Pamela Stanley, PhD; Ajit Varki, MD; Christopher Scott, PhD; Geert-Jan Boons, PhD; Salem Chouaib, PhD; and Peng Wu, PhD.

Aviceda has assembled an extraordinary multi-disciplinary team of world-class scientists and renowned researchers to join our efforts in developing the next generation of glyco-immune therapeutics for the treatment of immune-dysfunction conditions, said Mohamed A. Genead, MD, Founder, CEO & President of Aviceda Therapeutics. Each individual offers a fresh perspective and unique strategic acumen that complements and strengthens the insights of our in-house leadership development team.

Prof. Scott, Aviceda Scientific Co-Founder, is Director of the Patrick G Johnston Centre for Cancer Research and Cell Biology at Queens University Belfast. He is internationally renowned for his work in development of novel approaches in the field of antibody and nanomedicine-based therapies for the treatment of cancer and other conditions. Prof. Scott has a background in both the pharmaceutical industry and academia and was a founding scientist of Fusion Antibodies Plc. Research in his laboratory is funded by agencies such as Medical Research Council, UK charities and various industrial sources. He also held a Royal Society Industrial Fellowship with GSK from 2012 to 2015 and won the Vice Chancellors Prize for Innovation in 2015 with his groups work on developing a novel Siglec targeting nanomedicine for the treatment of sepsis and other inflammatory conditions.

The novelty of Avicedas platform technology is its potential to affect immune responses associated with a wide range of disease states, many of which are currently unmet or underserved needs. I look forward to the continued development of Avicedas core technology and moving forward to clinical trials that will pave the way for truly disruptive therapeutic strategies to enter the clinic that will significantly impact and improve patients lives in the not-too-distant future, said Prof. Scott.

Avicedas Scientific advisory chairwoman, Prof. Stanley, is the Horace W. Goldsmith Foundation Chair; Professor, Department of Cell Biology; and Associate Director for Laboratory Research of the Albert Einstein Cancer Center, Albert Einstein College of Medicine, New York. She obtained a doctorate degree from the University of Melbourne, Australia, for studies of influenza virus, and was subsequently a postdoctoral fellow of the Medical Research Council of Canada in the laboratory of Louis Siminovitch, University of Toronto, where she studied somatic cell genetics. Prof. Stanleys laboratory is focused on identifying roles for mammalian glycans in development, cancer and Notch signaling. Among her many varied contributions, Prof. Stanleys laboratory has isolated a large panel of Chinese hamster ovary (CHO) glycosylation mutants; characterized them at the biochemical, structural and genetic levels; and used them to identify new aspects of glycan synthesis and functions. She serves on the editorial boards of Scientific Reports, Glycobiology and FASEB Bio Advances; she is an editor of the textbook Essentials of Glycobiology; and her laboratory is the recipient of grants from the National Institutes of Health. Prof. Stanley has received numerous awards, including a MERIT award from the National Institutes of Health, an American Cancer Society Faculty Research Award, the Karl Meyer Award from the Society for Glycobiology (2003) and the International Glycoconjugate Organization (IGO) Award (2003).

Working with Aviceda represents a unique opportunity to contribute to science at the cutting edge. Its pipeline contains a broad range of candidates that represents numerous first-in-class opportunities, said Prof. Stanley.

Prof. Varki is currently a distinguished professor of medicine and cellular and molecular medicine, Co-director of the Glycobiology Research and Training Center and Executive Co-director for the UCSD/Salk Center for Academic Research and Training in Anthropogeny at the University of California, San Diego; and an Adjunct Professor at the Salk Institute for Biological Studies. Dr. Varki is also the executive editor of the textbook Essentials of Glycobiology. He received basic training in physiology, medicine, biology and biochemistry at the Christian Medical College, Vellore, The University of Nebraska, and Washington University in St. Louis, as well as formal training and certification in internal medicine, hematology and oncology. Dr. Varki is the recipient of numerous awards and recognitions, including election to the American Academy of Arts and Sciences and the US National Academy of Medicine, a MERIT award from the National Institutes of Health, an American Cancer Society Faculty Research Award, the Karl Meyer Award from the Society for Glycobiology and the International Glycoconjugate Organization (IGO) Award (2007).

The Aviceda team is already building on the foundational work in the emerging field of glycobiology to develop potential therapeutics and interventional strategies. Their work could be critically important for growing the understanding of how glycobiology and glycochemistry are applicable to immunology, and more broadly, to the field of drug and therapeutic development, said Prof. Varki.

Prof. Boons is a Distinguished Professor in Biochemical Sciences at the Department of Chemistry and the Complex Carbohydrate Research Center (CCRC) of the University of Georgia (USA) and Professor and Chair of the Department of Medicinal and Biological Chemistry of Utrecht University (The Netherlands). Prof. Boons directs a research program focused on the synthesis and biological functions of carbohydrates and glycoconjugates. The diversity of topics to which his group has significantly contributed includes the development of new and better methods for synthesizing exceptionally complex carbohydrates and glycoconjugates. Highlights of his research include contributions to the understanding of immunological properties of complex oligosaccharides and glycoconjugates at the molecular level, which is being used in the development of three-component vaccine candidates for many types of epithelial cancer; development of convergent strategies for complex oligosaccharide assembly, which make it possible to synthesize large collections of compounds with a minimal effort for structure activity relationship studies; and creation of a next generation glycan microarray that can probe the importance of glycan complexity for biological recognition, which in turn led to identification of glycan ligands for various glycan binding proteins that are being further developed as glycomimetics for drug development for various diseases. Among others, Prof. Boons has received the Creativity in Carbohydrate Science Award by the European Carbohydrate Association (2003), the Horace Isbell Award by the American Chemical Society (ACS) (2004), the Roy L. Whistler International Award in Carbohydrate

Chemistry by the International Carbohydrate Organization (2014), the Hudson Award (2015) and the Cope Mid-Career Scholar Award from ACS (2016).

Aviceda is leading the field of glycoimmunology in exciting new directions. I look forward to working with the company as it pursues multiple lines of development efforts that will someday transform the way immune-inflammatory conditions are treated in the clinic, said Prof. Boons.

Prof. Chouaib is the Director of Research, Institute Gustave Roussy, Paris, where he is active in research in tumor biology. Previously, Prof. Chouaib worked at the French National Institute of Health and Biomedical Research (INSERM) where he led a research unit focused on the investigation of the functional cross talk between cytotoxic cells and tumor targets in the context of tumor microenvironment complexity and plasticity. His research was directed at the transfer of fundamental concepts in clinical application in the field of cancer vaccines and cancer immunotherapy. Prof. Chouaib is a member of the American Association of Immunologists, New York Academy of Sciences, French Society of Immunologists, International Cytokine Society, American Association for Cancer Research, International Society for Biological Therapy of Cancer and American Association of Biological Chemistry. He was awarded the cancer research prize of the French ligue against cancer in 1992 and in 2004 the presidential prize in biotechnology. He was awarded for translational research and scientific excellency by INSERM. His research has resulted in more than 310 scientific articles and several reviews in the field of human immunology, tumor biology and cancer immunotherapy; he has also been an editor for several textbooks.

Dr. Wu is an Associate Professor in the Department of Molecular Medicine at Scripps Research. The current research in the Wu laboratory integrates synthetic chemistry with glycobiology to explore the relevance of protein glycosylation in human disease and cancer immunotherapy. In 2018, Dr. Wu developed a platform to construct antibody-cell conjugates for cancer immunotherapy, which does not require genetic engineering. Previously, while working as a postdoctoral fellow in the group of Professor Carolyn R. Bertozzi at the University of California, Berkeley, Dr. Wu developed an aldehyde-tag (SMARTag) based technology for site-specific labeling of monoclonal antibodies, which served as the foundation for Redwood Biosciences Inc., a biotech company co-founded by Bertozzi. In 2014, Redwood Bioscience Inc. and the SMARTag Antibody-Drug Conjugate technology platform was acquired by Catalent Pharma Solutions.

About Aviceda Therapeutics

Founded in 2018 and based in Cambridge, Massachusetts, Aviceda Therapeutics is a late-stage, pre-clinical biotechnology company with a mission to develop the next generation of glyco-immune therapeutics (GITs) utilizing a proprietary technology platform to modulate the innate immune system and chronic, non-resolving inflammation. Aviceda has assembled a world-class, cross-disciplinary team of recognized scientists, clinicians and drug developers to tackle devastating ocular and systemic degenerative, fibrotic, oncologic and immuno-inflammatory diseases. At Aviceda, we exploit a unique family of receptors found expressed on all innate immune cells and their associated glycobiological interactions to develop transformative medicines. Combining the power of our biology with our innovative cell-based high-throughput screening platform and proprietary nanoparticle technology, we can modulate the innate immune response specifically and profoundly. Aviceda is developing a pipeline of GITs that are delivered via biodegradable nanoparticles and which safely and effectively target numerous immune-inflammatory conditions. Avicedas lead ophthalmic optimized nanoparticle, as an intravitreal formulation, AVD-104, is being developed to target various immune system responses that contribute to pathology associated with age-related macular degeneration (AMD).

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Philadelphia Based Company Wants to Bring Back the Dead With Stem Cells – Gilmore Health News

October 31st, 2020 5:50 pm

Waking up the dead science fiction or a Halloween night horror movie? No, thats the goal of Bioquarks ReAnima project. The project aims to restore neuronal activity in brain dead people by combining several techniques: stem cell injection, nerve stimulation, and laser.

Stem Cells

Stem cells are increasingly becoming a serious treatment option for many nervous disorders: Alzheimers, Parkinsons, brain injuries, etc. So why not repair the brains of the dead to bring them back to life? This idea, worthy of a science fiction (or horror) film, is the crazy project of a company based in Philadelphia: Bioquark.

Read Also: Old Human Cells Successfully Rejuvenated Via Stem Cell Technology

This is not the first time that the company wants to participate in such an experiment. In 2016, the ReAnima study was launched in Bangalore, India, together with Himanshu Bansal, an orthopedic surgeon at Anupam Hospital. His plan was to combine several techniques to revive 20 brain dead people.

ReAnima consisted of injecting patients with mesenchymal stem cells and peptides that help regenerate brain cells; these peptides were to be supplied by Bioquark. In addition to these injections, transcranial laser stimulation and nerve stimulation were planned. This project was stopped by the Indian authorities in November last year, as revealed then by Science magazine.

But the company did not admit defeat. This time, according to the company, they are close to finding a new location for their clinical trials. Ira Pastor, CEO of Bioquark, told the Stat website that the company would announce the process in Latin America in the coming months.

Read Also: HGH Improves Memory In Stroke Victims Study Shows

If the experiment follows the same protocol as planned in India, it may involve 20 people. The clinical trial would again involve the injection of the patients stem cells, fat, blood Then a mixture of peptides would be injected into the spinal cord to stimulate the growth of new nerve cells. This compound, called BQ-A, was tested on animal models with head trauma. In addition, the nerves would be stimulated by nerve stimulation and 15 days of laser therapy to stimulate the neurons to make nerve connections. Researchers could then monitor the effects of this treatment using electroencephalograms.

But such a protocol raises many questions: How would a clinical trial be conducted on officially deceased people? If the person recovers some brain activity, in what state would he be? Will families be given false hope with a treatment that may take a long time?

Read Also: UC San Diego: Adult Brain Cells Revert to Younger State Following Injury, Study Shows

There is no indication that such a protocol will work. The company has not even tested the entire treatment on animal models! The mentioned treatments, such as injection of stem cells or transcranial stimulation, were tested in other situations, but not in cases of brain death. In an article published in 2016, neurologist Ariane Lewis and bioethicist Arthur Caplan stressed that the experiment had no scientific basis and that it gave families false and cruel hopes of a cure.

Experiment to raise the dead blocked in India

Response to a trial on reversal of Death by Neurologic Criteria

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Here’s What Happens When Lab-Grown Mini-Lungs Are Exposed to SARS-CoV-2 – ScienceAlert

October 31st, 2020 5:50 pm

Scientists working on a lab-grown mini-lung are now using their living model to better understand the current pandemic and potential new treatments.

The most recent version of this unique organoid is based entirely on human stem cells, known to repair the deepest parts of our lungs. When the researchers exposed it to SARS-CoV-2, the results were illuminating.

Dropping just one of these self-renewing units into a dish containing a tailored growth solution can produce millions of cells in a clump that resembles the tiny air sacs in human lungs.

Known as alveoli, these balloon-like sacs have shown diffuse damage in fatal cases of COVID-19, and while this havoc is often attributed to a storm of immune cells called cytokines, we're still figuring out how lung injury actually comes about.

The new mini-model gives us a glimpse of the battle on a molecular scale, and while it's nowhere near as complex as a real human lung, that's also what makes it easier to control and observe.

The unique organoid includes just one type of lung stem cell, known as an alveolar type 2 epithelial cell (AT2), which has the ability to self-renew, differentiate into other lung cells, keep the sac open with surfactants, and directly bind to viruses.

When the SARS-CoV-2 virus was introduced into this organoid's dish, researchers say the virus quickly infected the AT2 cells and spread throughout the alveoli-like structure.

The infection also triggered an inflammatory response in the organoid, reducing the production and proliferation of surfactant and inducing cell death, sometimes in surrounding areas that hadn't even yet been touched by the virus.

"This is a major breakthrough for the field because we were using cells that didn't have purified cultures," explains Ralph Baric, an epidemiologist, microbiologist, and immunologist at the University of North Carolina.

"This is incredibly elegant work to figure out how to purify and grow AT2 cells in culture."

Analysing the gene expression of these mini-organs, researchers found the inflammatory state triggered by the SARS-CoV-2 infection led to the production of interferons, cytokines, chemokines, and activation of genes related to cell death.

What's more, these signatures showed "striking similarity" to what's seen in severe COVID-19 patients.The results also match recent growing evidence that suggests severe cases of COVID-19 trigger a cytokine storm that may leave the lungs susceptible to damage.

Most of these observations, however, come from autopsies and have not been observed in living tissue.

This newly-developed model is a unique and versatile new way to study respiratory viruses in action, and it shows how a cascade of defences within stem cells themselves may cause more damage than good.

"It was thought cytokine storm happened due to the large influx of immune cells, but we can see it also happens in the lung stem cells themselves," says cell biologist Purushothama Rao Tata from Duke University.

"Now we have a way to figure out how to energise the cells to fight against this deadly virus," he adds.

In another set of experiments on the mini-lung, researchers found that administering low doses of interferons before infection slowed the spread of the virus, whereas reducing interferons before infection worsened the damage.

This suggests interferons are somehow mediating the immune response in our alveoli, slowing the cascade of cell death as the lung tries to get ahead of the infection.

But this may not be the whole picture; it's just a small insight into what's going on.Other recent studies show that while interferons might be a helpful treatment at certain stages of infection, at other times they can make matters worse.

While there are still many kinks and details that need to be ironed out in their model, researchers hope they can one day grow mini-lungs on which hundreds of experiments can be run at the same time, allowing us to figure out how the lung responds to infection and also how we can best protect it.

There's never been a more important time to learn more.

The study was published in Cell Stem Cell.

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Opinion: Proposition 14 Could Save the Life of Someone You Love – Times of San Diego

October 31st, 2020 5:50 pm

Share This Article:Embryonic stem cells. Image by Prue Talbot / UC RiversideBy Dr. Larry Goldstein

A yes vote on Proposition 14 is crucial to continue the pace of medical research and our states journey to save lives. For millions of Californians who live with a chronic disease or condition, and who need new therapies, this may be their last hope. Advancing medical progress to fight devastating and life-threatening diseases and conditions is an urgent matter now.

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Proposition 14 will continue funding for the California Institute for Regenerative Medicine, the states stem cell research funding institute. This institute is advancing medical discoveries and treatments for dozens of life-threatening or chronic diseases and conditions, including cancer, diabetes, heart disease, Alzheimers, Parkinsons, COVID-19 and more.

It is important to understand that the federal government and the private sector wont adequately fund the development of many important stem cell therapies. While the federal government has a strong focus on early lab research, it has also effectively banned funding of many important types of stem cell research and is threatening to ban more. On the other end, private funders have different priorities when it comes to funding medical research they almost exclusively invest in late-stage clinical trials where they can profit faster at lower financial risk.

There is a glaring funding gap between early lab work and late-stage clinical trials known as The Valley of Death that often ends promising stem cell research. With the sole mission of advancing the most promising treatments and cures, the institute bridges this critical gap, ensuring that potential life-changing therapies are not left stranded. The institutes unique approach is driving our state to achieve more progress, much faster, than we could have imagined.

The institutes funding has catalyzed or funded more than 90 clinical trials, two FDA-approved cancer treatments, and nine new treatments that have been designated as breakthrough therapies and have been fast-tracked for FDA approval. These breakthroughs will potentially help patients with cancer, diabetes, kidney disease, blindness, spinal cord injuries and immunodeficiencies.

While most of the 90+ clinical trials are still underway, many lives appear to have already been saved or improved. Babies born without immune systems are now surviving as are cancer patients who have exhausted all other treatment options. The institute has funded projects to help patients with Type I Diabetes produce their own insulin, blind patients start to regain eyesight, and quadriplegics start to regain upper body function.

All of these treatments in the pipeline if and when approved will also lead to spin-off treatments because they are fundamentally changing our knowledge and approach to treating chronic diseases. For example, by saving the lives of babies born with fatal immune disorders, the new knowledge and technologies are now being applied to treat other types of disorders. Cancer therapies being developed are effectively treating a handful of cancers today, but the knowledge and technology created can be applied to treating many other forms of cancer.

If Californians do not pass Proposition 14, our journey ends here many discoveries could be left on the shelf, delaying lifesaving and life-changing treatments for years.

Furthermore, as our state recovers from the impacts of COVID-19, Proposition 14 will provide an economic stimulus it will generate additional tax revenue and create many new jobs, and it wont cost the state anything until 2026. At a time when the cost of treating chronic diseases is straining our state budget and California families, Proposition 14 seeks to fund crucial disease research at a cost of less than a fraction of 1% of what Californians spend on chronic disease annually.

Close to 100 patient advocate organizations, major chambers of commerce across the state, Gov. Gavin Newsom, federal, state and local elected officials and the University of California Regents support Proposition 14. They do so because of its promise for therapy development, new business creation, and long-term financial benefit to all Californians.

We should remember that one of Californias core strengths is innovation and creation new types of businesses, new types of scientific research, and new ways to treat chronic diseases, conditions and illnesses. We cant afford to turn our back on these important goals. Our future depends on it. I hope you join me in voting YES on Proposition 14.

Dr. Larry Goldstein is a distinguished professor on the staff of the Shiley-Marcos Alzheimers Disease Research Centerat UC San Diego.

Opinion: Proposition 14 Could Save the Life of Someone You Love was last modified: October 31st, 2020 by Editor

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Cell Viability Assays and Consumables Market to Remain Lucrative During 2018 2025 – Aerospace Journal

October 31st, 2020 5:50 pm

The global market for cell viability assays should grow from $2.7 billion in 2018 to reach $4.1 billion by 2023 at a compound annual growth rate (CAGR) of 8.3% for the period of 2018-2023.

Report Scope:

The scope of this report is broad and covers various types of products available in the cell viability assays market and potential application sectors in various industries. The cell viability assays market is broken down by product into instruments and consumables. Revenue forecasts from 2018 to 2023 are given for each product, application, cell type and end user, with estimated valued derived from the revenues of manufacturers. Revenue generated from the installation and maintenance of instruments has been excluded from the report.

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The report also includes a discussion of the major players in each regional cell viability assays market. Further, it explains the major drivers and regional dynamics of the global market and current trends within the industry.

The report concludes with a special focus on the vendor landscape and includes detailed profiles of the major players in the global cell viability assays market.

Report Includes:

95 data tables and 66 additional tables An overview of the global cell viability assays market Analyses of global market trends, with data from 2017, 2018, and projections of compound annual growth rates (CAGRs) through 2023 Information on various type of products available in the cell viability assays market and potential applications across various industries Examination of the main product applications and markets in an effort to help companies and investors prioritize product opportunities and strategic movements Evaluation of key industry and market trends, and quantification of the main market segments, allowing the reader to better understand the industrys structure and changes occurring within it Coverage of innovations and development in stem cell research, toxicity testing and tissue engineering Insight into regulatory framework and investment analysis in the healthcare sector Detailed profiles of the major players of the industry, including Bio-Rad Laboratories Inc., Danaher Corp., GE Healthcare, Merck KGaA and R&D Systems Inc.

Summary

Cell viability assays refer to a homogeneous method to determine the number of viable cells within a culture. Cell viability measurements are used to evaluate the effectiveness of a drug candidate, rejection of implanted organs or evaluation of the life or death of cancerous cells. Cell viability assays are used in clinical and diagnostic applications, drug discovery and development, stem cell research, basic research and other applications such as toxicity testing and tissue engineering. Cell viability assays have proven to be extremely beneficial in drug discovery applications and the global cell viability assays market is projected to see rapid growth during the forecast period (2018-2023) owing to increasing emphasis on stem cell research and increasing demand for cell-based assays in research and development.

A surge in the number of potential biomarkers candidates for cell-based assays, growing incidence of infectious and chronic diseases and increasing focus on developing cell-based therapeutics are some of the major factors that are expected to promote the growth of global cell viability assays market. In addition, factors such as increasing healthcare expenditures and the global aging population are also providing traction for the global cell viability assays market growth during the forecast period. The growing prevalence of infectious and chronic diseases is considered to be the major driver behind the growth of the cell viability assays market. Cell viability measurements are used to evaluate theeffectiveness of a drug candidate, rejection of implanted organs or the life or death of cancerous cells.

According to the World Health Organization (WHO), the number of patients suffering from chronic diseases such as respiratory disease, cancer and cardiovascular disease totaled about 130 million people in 2005 and is expected to reach REDACTED by 2030. In addition, increasing emphasis on stem cell research is also poised to generate strong growth opportunities for cell viability assays. Governments globally are investing huge amounts on stem cell research. According to the U.S. Department of Health and Human Services, REDACTED billion was spent on stem cell research in 2016; this increased to REDACTED in 2017. In April 2017, Bayer AG and Versant Ventures (U.S.) announced plans to invest REDACTED to launch a stem cell research company in Canada and create a global hub for regenerative-medicinetherapies.

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The global cell viability assays market is projected to rise at a compound annual growth rate (CAGR) of REDACTED during the forecast period of 2018 through 2023. Market value is expected to rise from REDACTED in 2018 to REDACTED by 2023. Consumables held REDACTED of the market in 2017 in terms of revenue. By 2023, total revenue from consumables is expected to see a CAGR of REDACTED.

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Humanigen Announces First Patient Dosed at MedStar Washington Hospital Center in Phase 3 Clinical Study of Lenzilumab in COVID-19 – Business Wire

October 31st, 2020 5:50 pm

BURLINGAME, Calif.--(BUSINESS WIRE)--Humanigen, Inc. (HGEN) (Humanigen), a clinical stage biopharmaceutical company focused on preventing and treating an immune hyper-response called cytokine storm with lenzilumab, today announced that MedStar Washington Hospital Center in Washington, D.C. treated its first COVID-19 patient with lenzilumab. The primary goal of this Phase 3 randomized, double-blind, multicenter, placebo-controlled clinical trial is to determine if lenzilumab can help hospitalized patients with COVID-19 recover faster. As many as 89% of hospitalized patients with COVID-19 are at risk of a complication called cytokine storm, a harmful inflammation that has been the leading cause of COVID-19 death. MedStar Washington Hospital Center is one of 18 sites in the U.S. approved to enroll eligible patients to study lenzilumab, designed specifically to stop this storm. Eligible patients can participate in this trial while also receiving other standard-of-care therapies as recommended by their treating physician.

Given the growing number of cases in the D.C. area seen in the past few weeks, we were particularly motivated to ensure our Phase 3 study was enrolling and accessible, said Cameron Durrant, MD, MBA, chief executive officer of Humanigen. We have been impressed with the hospital leadership and trial investigators at MedStar Washington, and worked together with speed and efficiency to get this trial location ready to enroll patients.

For more information on participating in the Phase 3 COVID-19 trial of lenzilumab, please visit StopStorm.com, and talk to your doctor to see if you may be eligible to participate.

More details on Humanigens programs in COVID-19 can be found on the Companys website at http://www.humanigen.com under the COVID-19 tab, and details of the U.S. Phase 3 potential registration study can be found at clinicaltrials.gov using Identifier NCT04351152.

About Humanigen, Inc.

Humanigen, Inc. is developing its portfolio of clinical and pre-clinical therapies for the treatment of cancers and infectious diseases via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms. We believe that our GM-CSF neutralization and gene-editing platform technologies have the potential to reduce the inflammatory cascade associated with coronavirus infection. The companys immediate focus is to prevent or minimize the cytokine release syndrome that precedes severe lung dysfunction and ARDS in serious cases of SARS-CoV-2 infection. The company is also focused on creating next-generation combinatory gene-edited CAR-T therapies using strategies to improve efficacy while employing GM-CSF gene knockout technologies to control toxicity. In addition, the company is developing its own portfolio of proprietary first-in-class EphA3-CAR-T for various solid cancers and EMR1-CAR-T for various eosinophilic disorders. The company is also exploring the effectiveness of its GM-CSF neutralization technologies (either through the use of lenzilumab as a neutralizing antibody or through GM-CSF gene knockout) in combination with other CAR-T, bispecific or natural killer (NK) T cell engaging immunotherapy treatments to break the efficacy/toxicity linkage, including to prevent and/or treat graft-versus-host disease (GvHD) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Additionally, Humanigen and Kite, a Gilead Company, are evaluating lenzilumab in combination with Yescarta (axicabtagene ciloleucel) in patients with relapsed or refractory large B-cell lymphoma in a clinical collaboration. For more information, visit http://www.humanigen.com.

Forward-Looking Statements

This release contains forward-looking statements. Forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct and you should be aware that actual events or results may differ materially from those contained in the forward-looking statements. Words such as "will," "expect," "intend," "plan," "potential," "possible," "goals," "accelerate," "continue," and similar expressions identify forward-looking statements, including, without limitation, statements regarding our expectations for the Phase 3 study and the potential future development of lenzilumab, our pathway to our intended submission for, and potential receipt of, an Emergency Use Authorization and potential subsequent BLA from FDA, and statements regarding the potential for lenzilumab to be used to prevent or treat GvHD and, as sequenced therapy with Kites Yescarta, in CAR-T therapies. Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to, the risks inherent in our lack of profitability; our dependence on partners to further the development of our product candidates; the costs and the uncertainties inherent in the development and launch of any new pharmaceutical product; the outcome of pending or future litigation; and the various risks and uncertainties described in the "Risk Factors" sections and elsewhere in the Company's periodic and other filings with the Securities and Exchange Commission.

All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You should not place undue reliance on any forward-looking statements, which speak only as of the date of this release. We undertake no obligation to revise or update any forward-looking statements made in this press release to reflect events or circumstances after the date hereof or to reflect new information or the occurrence of unanticipated events, except as required by law.

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Penn Medicine Researchers Receive $5.4 million Grant to Find Genetic Drivers of Testicular Cancer – Newswise

October 31st, 2020 5:49 pm

Newswise PHILADELPHIAThe international hunt to find more genetic risk markers for testicular cancer is expanding. A team of researchers led by Katherine L. Nathanson, MD, deputy director of the Abramson Cancer Center and the Pearl Basser Professor for BRCA-Related Research in the Perelman School of Medicine at the University of Pennsylvania, was recently awarded $5.4 million over five years from the National Institutes of Health to continue the long-standing genomics work of the TEsticular CAncer Consortium (TECAC).

A total of nearly $7 million has been awarded to TECAC, which includes researchers from 27 institutions around the world, whose collaborative goal is understand the genetic susceptibility to testicular germ cell tumors (TGCT).

TGCT are the most common cancer in the United States and Europe in men between the ages of 15 to 45, and the number of cases has continued to rise over the past 40 years. Approximately 50 percent of the risk of disease is due to genetic factors, higher than for other cancer types.

To date, TECAC has identified 22 novel susceptibility alleles, bringing the total number of risk markers to 66. Nathanson led a study in 2017 published in Nature Genetics that identified eight of those markers in previously unknown gene regions, as well as four in previously identified regions.

Members of TECAC also were the first to identify CHEK2, a moderate penetrance gene for TGCT. Penetrance refers to the proportion of people with a mutation in specific gene. Unlike other solid tumor types (e.g. breast, ovarian), the inherited risk of TGCT is likely due to multiple variants rather than any single gene.

Our work has revealed critical roles for genetic variants and mutations in testicular germ cell tumors and defined the biology of TGCTs as associated with defects in maturation of male germ cells, but theres still much more to discover with this highly heritable disease, Nathanson said. This grant will allow us to continue to pool our resources and expertise to better understand its biology and etiology, as well as provide data that can help identify men at higher risk of the disease and in need of surveillance.

The latest round of funding will focus on three projects: identify rare and common variants using whole exome genetic sequencing from biosamples of more than 2,000 men; conduct a transcriptome-wide association study, or TWAS, to identify novel candidate susceptibility genes in nearly 250,000 men (the largest to date); and further evaluate any variants or gene discovered from those two projects using tools, such as CRISPR, in cells.

Other Penn collaborators on this grant (R01 CA164947 A1) include David Vaughn, Linda Jacobs, Li-San Wang and Mingyao Li.

##

Penn Medicineis one of the worlds leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania(founded in 1765 as the nations first medical school) and theUniversity of Pennsylvania Health System, which together form a $8.6 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $494 million awarded in the 2019 fiscal year.

The University of Pennsylvania Health Systems patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Centerwhich are recognized as one of the nations top Honor Roll hospitals byU.S. News & World ReportChester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nations first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 43,900 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2019, Penn Medicine provided more than $583 million to benefit our community.

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enGene Receives Funding Through Cystic Fibrosis Foundation’s Path to a Cure for the Discovery of Novel Gene Therapies to Treat Cystic Fibrosis -…

October 31st, 2020 5:49 pm

BOSTON and MONTRAL, Oct. 29, 2020 /PRNewswire/ -enGeneInc.,abiotechnology company developing non-viral gene therapies for local administration into mucosal tissues enabled by its proprietary DDX platform, announced today an award from the Cystic Fibrosis Foundation for the discovery of genetic medicines to treat patients with cystic fibrosis (CF).

The award was made as a part of the CF Foundation's $500 million Path to a Cure initiative to accelerate the discovery and development of treatments that address the underlying cause of the disease.

Affecting over 75,000 patients worldwide, CF is a genetic disease caused by mutations in a gene known as the cystic fibrosis transmembrane conductance regulator (CFTR) that render a non-functional CFTR protein. Consequently, multiple organs are affected by disease, chief among them the lungs, where chronic infections and a worsening ability to breathe leads to progressive lung damage and premature death. Patients with nonsense and other rare mutations in both copies of the CFTR gene currently have no therapies that treat the underlying cause of the disease.

"Gene therapy holds promise for the treatment of CF by delivering a functional copy of the CFTR gene to the lungs to restore function and alleviate disease. enGene is developing a DDX-based inhalable formulation to carry DNA to the airways with the goal of functional complementation of CFTR mutations. We are thrilled to have the support of the Cystic Fibrosis Foundation to discover novel gene therapy candidates for patients with CF," commented Jose Lora, CSO of enGene.

In developing an inhalable gene therapy for CF, enGene is coupling a non-viral DNA payload to its biocompatible DDX carrier in an effort to create genetic medicines that allow repeatable and titratable dosing to achieve meaningful efficacy.

"Gene therapies have made a remarkable impact in many fields of medicine, but unlocking their full potential in mucosal tissues such as the lung has been elusive, leaving many patients with CF without available treatment options. We are honored to be working with the CF Foundation to accelerate our research and development efforts towards improving and extending the lives of all CF patients," said Jason Hanson, enGene's President and CEO.

About enGene Inc.enGene Inc. is a biotechnology company developing a proprietary non-viral gene therapy platform for localized delivery of nucleic acid payloads to mucosal tissues. The dually derived chitosan (DDX) platform has a high-degree of payload flexibility including DNA and various forms of RNA with broad tissue and disease applications. In addition to developing gene therapies for the lungs, enGene has developed a unique dual-immune activator for patients with non-muscle invasive bladder cancer which has completed IND-enabling studies. The company is evolving its technology to enable applications in multiple mucosal tissues with areas of high unmet medical need.www.engene.com/

Note regarding forward-looking statementsThis press release contains certain "forward-looking statements" that reflect the Company's beliefs and assumptions based on currently available data and information. These forward-looking statements fall within the meaning of the "safe harbor" provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "target," "believe," "expect," "will," "may," "anticipate," "estimate," "would," "positioned," "future," and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on enGene's current beliefs, expectations, and assumptions that by definition involve risks, uncertainties, that are difficult to predict and are subject to factors outside of management's control and that could cause actual results to differ substantially from statements made including but not limited to: risks associated with the success of preclinical studies, clinical trials, research and development programs, as well as regulatory approval processes. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. enGene has no approved drugs available for sale marketing at this time and may never have an approved drug. You are cautioned not to rely on enGene's forward looking statements, which are only made as of the date hereof. The Company is under no obligation to update these statements.

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http://engene.com/

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Prowess of Bionano Genomics’ Saphyr System in Uncovering Novel Genetic Variations That Cause Cancer and Genetic Disease in Full Display at ASHG 2020 -…

October 31st, 2020 5:49 pm

SAN DIEGO, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) announced that human genetics researchers using the Saphyr system will present their results at the American Society of Human Genetics (ASHG) Annual Meeting, being held virtually at http://www.ashg.org between October 27-30. The impact of structural variation analysis using the Saphyr system will be demonstrated at ASHG with 18 oral and poster presentations which cover an expanding array of diseases like cancer predisposition, microdeletion syndromes, repeat expansion disorders, neurodegenerative diseases, disorders of sex development and a variety of other genetic diseases. Additionally, these presentations show Saphyrs abilities to elucidate the exact structure of complex genomic rearrangements such as large inversions, chromothripsis and low copy repeats.

The scientific importance and quality of the studies utilizing Saphyr and presented at ASHG have increased year over year, said Erik Holmlin, Ph.D., CEO of Bionano. As more scientists present and publish their important discoveries made with Saphyr, an increasing number of potential future Saphyr users become aware of its prowess in uncovering novel genetic variants that contribute to cancer and genetic disease, which could drive more adoption and utilization for basic genetic research and clinical studies alike.

Below is a summary of key presentations to be given at ASHG 2020 featuring the use of Bionanos optical genome mapping technology:

Live Presentation October 29, 2020, 11:45AM-12:00PMDeciphering Genomic InversionsChristopher M. Grochowski, Baylor College of MedicineGenomic inversions are a class of structural variation (SV) relevant in evolution, speciation, and human disease but challenging to detect and resolve using current genomic assays. While short-read WGS can detect a fraction of copy number neutral inversions, those mediated by repeats or accompanied by CNVs remain challenging. The utilization of multiple technologies and visualization of unbroken DNA through long molecule approaches facilitate detection ofin cisevents and resolution of SVs containing two or more breakpoint junctions.

The following Co-Labs, Poster Sessions and Abstracts are available for on-demand viewing during and after ASHG 2020:

Bionano Laboratory Co-Lab Session: Resolving Complex Haplotypes Implicated in Alzheimers and Other Neurodegenerative Diseases.Mark T. W. Ebbert, Neuroscience Department, Mayo ClinicAlzheimers disease is genetically complex with no meaningful therapies or pre-symptomatic disease diagnostics. Most of the genes implicated in Alzheimers disease do not have a known functional mutation, meaning there are no known molecular mechanisms to help understand disease etiology.

In this co-lab session, Mark T. W. Ebbert of the Mayo Clinic will discuss his teams work toward identifying functional structural mutations that drive disease in order to facilitate a meaningful therapy and pre-symptomatic disease diagnostic. Some of the genes and regions implicated in Alzheimers disease are genomically complex and cannot be resolved with short-read sequencing technologies. These regions include MAPT, CR1, and the histocompatibility complex (including the HLA genes).

3342 Bionano Poster Session: High Throughput Analysis of Disease Repeat Expansions and Contractions by Optical MappingErnest Lam, Sr Manager Bioinformatics, Bionano GenomicsRepeat expansions and contractions are associated with degenerative disorders such as facioscapulohumeral muscular dystrophy (FSHD). Southern Blotting is the gold standard for long repeat analysis but has many limitations. Optical genome mapping allows for efficient analysis of diseases associated with repeat expansion and contraction.

2190 Bionano Poster Session: Rapid Automated large Structural Variation Detection in Mouse Genome by Whole Genome SequencingJill Lai, Sr Applications Scientist, Bionano GenomicsIdentifying SVs for key model organisms such as mouse and rat is essential for genome interpretation and disease studies but has been historically difficult due to limitations inherent to available genome technologies. We updated the Saphyr analysis pipeline such that copy number variant (CNV) and SV analyses could now be applied to mouse and other non-human species, and constructed a control SV database for annotating variants, and identified strain-specific SVs/CNVs as well as variation shared among strains.

Additional presentations/abstracts featuring optical genome mapping:

3208 - Long-read sequencing and optical mapping decipher structural composition ofATXN10repeat in kindred with spinocerebellar ataxia and Parkinsons diseasePresented by Birgitt Schuele, Associate Professor, Department of Pathology, Stanford University School of Medicine

3270 - Uniparental isodisomy, structural and noncoding variants involved in inherited retinal degeneration (IRD) in three pedigreesPresented by Pooja Biswas, Ophthalmology Department, University of California, San Diego

Data CoLab: Whole Genome Map Assembly and Structural Variation Analysis with Hitachi Human Chromosome ExplorerPresented by Hitachi-High-Tech America, Inc.

2123 - High-throughput sequencing and mapping technologies applied to 10 human genomes with chromothripsis-like rearrangementsPresented by Uir Souto Melo, Mundlos Lab, Max Planck Institute for Molecular Genetics, Berlin, Germany

2165 -nanotatoR: A tool for enhanced annotation of genomic structural variantsPresented by Emmanuele Delot, Center for Genetic Medicine Research, Childrens National Hospital, Washington, DC

2998 - Highly variable structure and organization of the human 3q29 subtelomeric segmental duplicationsPresented by Umamaheswaran Gurusamy, Cardiovascular Research Institute, University of California San Francisco

2304 - Enlightening the dark matter of the genome: Whole genome imaging identifies a germline retrotransposon insertion inSMARCB1in two siblings with atypical teratoid rhabdoid tumorPresented by Mariangela Sabatella, Princess Mxima Center for Pediatric Oncology, Utrecht, Netherlands

2318 - FaNDOM: Fast Nested Distance-based seeding of Optical MapsPresented by Siavash Raeisi Dehkordi, Computer Science & Engineering, University of California San Diego, La Jolla

3023 - Structural hypervariability of low copy repeats on chromosome 22 is human specificPresented by Lisanne Vervoort, Department of Human Genetics, KU Leuven, Leuven, Belgium

3024 - Telomere-to-telomere assembly and complete comparative sequence analysis of the human chromosome 8 centromereReviewer's Choice Award RecipientPresented by Glennis Logsdon, Genome Sciences, University of Washington, Seattle, WA

3311 - Comprehensive structural variant identification with optical genome mapping and short-read sequencing for diagnosis of disorders/differences of sex development (DSD)Reviewer's Choice Award RecipientPresented by Hayk Barseghyan, Center for Genetic Medicine Research, Children's National Hospital, Washington, DC

3318 - De novo mutation and skewed X-inactivation in girl with BCAP31-related syndromePresented by H.J. Kao, Institute of Biomedical Sciences, Academia Sinica, Taipei, Taiwan

3560 - Resolving genomic structures inMECP2Duplication Syndrome provides insight into genotype-phenotype correlationsReviewer's Choice Award RecipientPresented by Davut Pehlivan, Molecular and Human Genetics, Baylor College of Medicine, Houston, TX

2157 -methometR: quantification of long-range haplotype specific methylation levels from Optical Genome MapsPresented by Surajit Bhattacharya, Center for Genetic Medicine Research, Childrens Research Institute, Childrens National Hospital, Washington, DC

About Bionano GenomicsBionano is a genome analysis company providing tools and services based on its Saphyr system to scientists and clinicians conducting genetic research and patient testing, and providing diagnostic testing for those with autism spectrum disorder (ASD) and other neurodevelopmental disabilities through its Lineagen business. Bionanos Saphyr system is a platform for ultra-sensitive and ultra-specific structural variation detection that enables researchers and clinicians to accelerate the search for new diagnostics and therapeutic targets and to streamline the study of changes in chromosomes, which is known as cytogenetics. The Saphyr system is comprised of an instrument, chip consumables, reagents and a suite of data analysis tools, and genome analysis services to provide access to data generated by the Saphyr system for researchers who prefer not to adopt the Saphyr system in their labs. Lineagen has been providing genetic testing services to families and their healthcare providers for over nine years and has performed over 65,000 tests for those with neurodevelopmental concerns. For more information, visitwww.bionanogenomics.com or http://www.lineagen.com.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as may, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the timing and content of the presentations identified in this press release; the effectiveness and utility of Bionanos technology in basic genetic research and clinical settings; the contribution of Saphyr to uncovering novel genetic variants that contribute to cancer and genetic disease; the benefits of Bionanos optical mapping technology and its ability to facilitate genomic analysis in future studies; and Bionanos strategic plans. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the risks and uncertainties associated with: the impact of the COVID-19 pandemic on our business and the global economy; general market conditions; changes in the competitive landscape and the introduction of competitive products; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of medical and research institutions to obtain funding to support adoption or continued use of our technologies; the loss of key members of management and our commercial team; and the risks and uncertainties associated withour business and financial condition in general, including the risks and uncertainties described in our filings with the Securities and Exchange Commission, including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2019 and in other filings subsequently made by us with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise.

CONTACTSCompany Contact:Erik Holmlin, CEOBionano Genomics, Inc.+1 (858) 888-7610eholmlin@bionanogenomics.com

Investor Relations Contact:Ashley R. RobinsonLifeSci Advisors, LLC+1 (617) 430-7577arr@lifesciadvisors.com

Media Contact:Darren Opland, PhDLifeSci Communications+1 (617) 733-7668darren@lifescicomms.com

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New screening tool could turn up genes tied to developmental disorders – STAT

October 31st, 2020 5:49 pm

Scientists in Vienna have developed a new human tissue screening technique that has identified previously unknown genes involved in causing microcephaly, a rare genetic disorder, and that could one day be used to identify unknown genes tied to other conditions.

In a study published Thursday in Science, researchers screened lab-grown human brain tissues for 172 genes thought to be associated with microcephaly, a condition in which babies are born with smaller-than-normal brains and have severe mental impairments. The search revealed 25 new genes linked to this rare neurological condition, adding to the 27 already known genes tied to microcephaly. The researchers also uncovered the involvement of certain pathways that were previously unknown to be connected to the disease.

This is a proof of concept, said Jrgen Knoblich, a molecular biologist at the Austrian Academy of Sciences Institute of Molecular Biotechnology and co-author of the study. With our ability to query many diseased genes at the same time and ask which ones are relevant in a human tissue, we can now study other diseases and other organs.

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For decades scientists have relied on small animals as models to make sense of how a human brain develops. But it turns out that our brains are not blown-up versions of a rodent brain. Mice and rat brain surfaces, for instance, are smooth, unlike the shrivelled walnut look of a human brain, with its countless folds. Also, these rodents are born with a somewhat complete brain, in which most neurons are in place, although they continue to form new connections after birth. In a human child, on the other hand, there are a massive number of neurons that form and populate the cortex after birth.

There are some processes that happen in our brain and not in mice brains that are responsible for human brains becoming so big and powerful, Knoblich said. This generates a very big medical problem, which is how do we study processes that are only happening in humans.

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To address this problem, several scientists including Knoblich developed human brain organoids that are no bigger than a lentil, created from stem cells, and function just like a working human brain. With an interest in studying neurodevelopmental disorders like microcephaly, Knoblichs team used these miniature substitute brains to look for clues about the genes that may hamper brain development.

Typically, scientists conduct genetic screening by inactivating select genes one by one to understand their contribution to bodily functions. But screens of human genes are restricted to cells grown in petri dishes in two dimensions, in which cells dont interact very much.

Microcephaly is a tissue disease and we couldnt really study it in 2D, said Christopher Esk, a molecular biologist at the Austrian Academy of Sciences Institute of Molecular Biotechnology and co-lead author of the study.

So, the researchers developed a technique called CRISPR-Lineage Tracing at Cellular resolution in Heterogeneous Tissue, which uses the gene-editing technology to make cuts in DNA and knockout genes in combination with a barcoding technology that tracks parent stems and their progeny cells as the 3D brain organoid develops.

Using an organoid developed from cells of a microcephalus patient, they kept an eye out for mutations that gave rise to fewer cells and thus a small brain in comparison with a healthy one.

The researchers used CRISPR-LICHT to simultaneously screen 172 potential microcephaly causing gene candidates and found 25 to be involved.

Among them was a gene called Immediate Early Response 3 Interacting Protein 1 in the endoplasmic reticulum, which is the protein processing station within a cell. This protein processing is required to properly process other proteins, among them extracellular matrix proteins, which are in turn important for tissue integrity, and thus brain size, Esk said.

Kristen Brennand, a stem cell biologist at the Icahn School of Medicine at Mount Sinai in New York, who wasnt involved in the study, said she appreciated how the research captured this causal link. Clinical genetics can identify mutations in patients, but fall short of identifying causal mutations that definitively underlie disease risk, she said.

Going forward, Knoblich and his colleagues hope to use CRISPR-LICHT to screen many more genes that may be associated with other brain development disorders. Weve done it for microcephaly, and were already doing it for autism, he said. But the method can be applied to any type of organoid or any type of disease and any cell type.

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