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The White House is hosting indoor holiday parties despite top health officials’ warnings | TheHill – The Hill

December 4th, 2020 12:28 am

The White House is scheduled to hold as many as 20 indoor holiday events this month despite an alarming surge in coronavirus cases and hospitalizations and guidance from public health officials to avoid large indoor gatherings this holiday season.

Leading up to Thanksgiving, the Centers for Disease Control and Prevention (CDC) issued guidance recommending people spend the holiday only with those living in the same household and suggestedcelebrations be held outdoors if possible as the coronavirus raged out of control.

Our country is in a historic fight against the Coronavirus. Add Changing America to your Facebook or Twitter feed to stay on top of the news.

During an interview just ahead of the holiday, U.S. Surgeon General Jerome Adams pleaded with Americans to avoid large holiday gatherings as the U.S. is at a dire point in the COVID-19 pandemic.

As of Wednesday, a record 98,691 COVID-19 patients were hospitalized across the country while the rolling seven-day average of deathshas surpassed 1,500.

But the White House has moved forward with annual holiday events as the outbreak continues, ignoring warnings from the Trump administrations own public health officials and a pandemic that has left more than 273,000 Americans dead.

The events kicked off Monday after first lady Melania Trump unveiled Christmas decorations.

The Washington Post reports each event will include more than 50 guests, most of whom will not be tested in advance. One such event on Tuesday night included several attendees who were not wearing masks and crowded together, according to NBC News.

White House press secretary Kayleigh McEnany on Wednesday dismissed the health concerns associated with the events.

If you can loot businesses, burn down buildings, engage in protest, you can also go to a Christmas party, McEnany said during a White House briefing.

You can celebrate the holiday of Christmas, and you can do it responsibly, she said.

Stephanie Grisham, Melania Trumps chief of staff, said in a statement on Wednesday that the White House is providing the safest environment possible by having a smaller guest list and mask requirements while encouraging social distancing.

Meanwhile, the head of the CDC warned the U.S. could see another 200,000 coronavirus deaths over the next three months if people dont take mitigation measures such as mask-wearing and social distancing seriously.

The reality is December and January and February are going to be rough times. I actually believe theyre going to be the most difficult in the public health history of this nation, CDC Director Robert Redfield said Wednesday.

WHAT YOU NEED TO KNOW ABOUT CORONAVIRUS RIGHT NOW

CDC CUTS LENGTH OF COVID-19 QUARANTINE TIME AFTER EXPOSURE

UK BECOMES FIRST WESTERN NATION TO AUTHORIZE COVID-19 VACCINE

CDC DECIDES WHO WILL RECEIVE FIRST DOSES OF COVID-19 VACCINES

CORONAVIRUS EPIDEMIC WAS SPREADING IN US LAST CHRISTMAS, LONG BEFORE IT WAS IDENTIFIED IN CHINA, NEW STUDY FINDS

FAUCI CONFIRMS COVID-19 VACCINE COULD BE READY BY LATE DECEMBER, EARLY 2021

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BioAge Raises $90M to Treat Aging and Age-related Diseases – GlobeNewswire

December 4th, 2020 12:28 am

Proceeds will support advancement of the companys pipeline and proprietary platform for identifying and developing therapies to treat diseases of aging

Company announces Chief Medical Officer as it prepares to enter clinic in 2021 with lead Phase 2-ready therapeutic compounds

RICHMOND, Calif., Dec. 03, 2020 (GLOBE NEWSWIRE) -- BioAge Labs, Inc., a biotechnology company developing medicines to treat aging and aging-related diseases, today announced that it has raised $90 million in an oversubscribed Series C financing. The raise was co-led by Andreessen Horowitz and serial entrepreneur, Elad Gil, and included new investors Kaiser Foundation Hospitals, AARP Foundation (through the RockCreek Impact Fund) and Phi-X Capital, the fund of genomics entrepreneur Mostafa Ronaghi, among others. Current investors including Caffeinated Capital, Redpoint Ventures, PEAR Ventures, AME Cloud Ventures, Felicis Ventures, and others also participated.

These additional funds will support advancement of our systems biology and data-driven platform to map the key pathways that drive human aging and our pipeline of medicines that target these pathways to reverse or eradicate diseases and extend healthspan, said Kristen Fortney, PhD, co-founder and chief executive officer. We look forward to advancing our first platform-derived therapies, BGE-117 and BGE-175 into clinical trials in the first half of 2021.

BioAge is at the forefront of understanding scientific drivers of aging with its unique and cutting-edge systems biology platform. We believe their approach has the potential to unlock the underlying pathologies of many diseases, such as Alzheimers, cardiovascular disease, and frailty, that disproportionately affect older populations, said Vijay Pande, PhD, general partner at Andreessen Horowitz.

Drugs that target aging have potential to treat several morbid diseases and improve the lives of older adults. BioAge has built a proprietary engine to analyze molecular signatures in aging populations, and to advance data-driven hypotheses to identify existing clinical-stage drugs that are ready for Phase 2 efficacy trials in age-related diseases. Im excited to work with them as they scale their platform and develop multiple therapies to improve the health of older individuals, said Elad Gil, PhD, entrepreneur and investor.

Proceeds from the financing will be used to build and develop a diversified portfolio of therapies that increase healthspan and lifespan, augment BioAges artificial intelligence (AI)-driven approach to map the molecular pathways that impact human longevity, and further expand capabilities to test drug candidates in predictive models of human diseases of aging.

Separately, the company announced that Paul Rubin, M.D. will lead its clinical development efforts as chief medical officer. Dr. Rubin, a veteran biopharmaceutical drug developer, brings extensive experience from early discovery, late stage development and ultimate drug approvals across a wide variety of therapeutic areas in global geographies. In former roles heading development at Sepracor, GSK and Abbott, Dr. Rubin has been responsible for the clinical development and approval of more than ten products. Prior to BioAge, he was most recently executive vice president, Research and Development for MiRagen and held a similar role at Xoma, two biotechnology companies. Dr. Rubin earned his M.D. at Rush Medical College, Chicago.

Dr. Rubin commented, I am thrilled to be leading the BioAge clinical development team at this exciting time. Our platform provides a unique opportunity to identify targets relevant to the aging process and diseases associated with this process. Diseases common to older patients are often ignored despite their direct association with morbidity, mortality and decreased quality of life. The first two drugs from our pipeline are ready to begin Phase 2 trials in indications targeting serious conditions in elderly populations that presently have no good therapeutic options. A key aspect of our strategy is to initiate efficient human clinical trials that will demonstrate that our drugs can address age-related deficiencies in acute conditions, which may expedite approval and serve as a gateway to the treatment of chronic diseases, resulting in healthy aging.

About the BioAge Platform

The BioAge platform identifies key drug targets that will impact aging. The companys proprietary human aging cohorts have blood samples collected up to 45 years ago, with participant -omics data that is tied to extensive medical follow-up records including detailed future healthspan, lifespan and disease outcomes. BioAge has built a systems biology and AI platform that leverages these rich datasets to identify the molecular drivers of age-related pathology. BioAges pipeline of therapies targeting these key pathways will address the significant unmet medical needs of an aging population.

About BioAge

BioAge is a privately-held biotechnology company developing proprietary drugs to treat aging and aging- related diseases. Since its founding in 2015, the Company has raised $127 million in venture capital funding to back its AI-driven approach to map the molecular pathways that impact human longevity. BioAges mission is to develop a pipeline of therapeutic assets that increase healthspan and lifespan. For additional information about BioAge, visit the companys website at http://www.bioagelabs.com.

Source: BioAge Labs, Inc.

Contact Information:

BioAge | peng@bioagelabs.com

Media | swheeler@wheelhouselsa.com

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How the pandemic enabled a robot revolution – Politico

December 4th, 2020 12:27 am

Future Pulse is a new weekly newsletter exploring the rapidly changing world of health care and technology. We call out fads from real advances, chronicle experimentation and investigate the tension between innovation, regulation and privacy. Join the conversation!

JUST WHAT THE ROBOT ORDERED: At several Texas health networks, a friendly-looking robot with a mechanical arm and blinking lights for eyes can be seen whirring around the hospital floor, ferrying lab samples and protective gear for nursing staff. At Bostons Brigham & Womens this summer, a roaming four-legged robot with mounted infrared cameras loped into the emergency department to take patients vital signs. At Los Angeles Adventist Health White Memorial, seven devices the size of mini-fridges zip around to disinfect patient rooms with UV light.

Whether theyre restocking supply rooms or taking patients heart rates, the roving machines could help contain the coronavirus spread by cutting down on possible exposure between staff and patients, health system leaders say. The systems were already in the early stages of piloting robots before the pandemic, but the crisis has generated more interest than ever, robotics experts tell us. The big question is whether to rush to automate is pushing aside viable existing technologies

What were trying to do is introduce ways of really preserving safety of health care personnel by having assistance ... in doing tasks that can essentially be handed off to a robot, says MIT assistant professor Giovanni Traverso, part of the research team that sent Boston Dynamics dog-like robot named Spot into Brigham & Womens exam rooms to measure vital signs like temperature and blood pressure.

Rich Pedroncelli/AP Photo

Stopped at the door: Still, there are lots of mundane challenges some robots havent been able to overcome, like walking up stairs or opening certain types of doors. For robots in general, the standard doorknob is still going to be most challenging, says Andrew Rossi of Diligent Robotics, which created the Moxi robot used in the Texas health systems.

And anytime the robots are deployed to a new health system, they have to be programmed with new instructions. We have to understand what elevators they use and how they badge in and out of doors and then we have to make adjustments, Rossi said.

Theres much to learn before health systems go all in with robots, MITs Traverso said. His team is exploring whether Boston Dynamics Spot, which retails for about $75,000, is worth the investment. Theyre also considering patients comfort levels with the robot caregivers, which Traverso admits might be a little jarring. In general, though,people are curious and kind of fascinated by this robot, he says.

A simpler approach?: And the biggest question remains is the high-tech way ultimately the better way?

I think we need to further expand our understanding and exposure to robots and see them in different settings, Traverso says. The main question is, do we need that level of sophistication, or are there simpler systems?

Welcome back to Future Pulse, where we explore the convergence of health care and technology. Share your news and feedback: @dariustahir, @ravindranize, @ali_lev, @katymurphy.

Next week, POLITICO will feature a special edition Future Pulse newsletter at the Milken Institute Future of Health Summit. The newsletter takes readers inside one of the most influential gatherings of global health industry leaders and innovators determined to confront and conquer the most significant health challenges. Covid-19 has exposed weaknesses across our health systems, particularly in the treatment of our most vulnerable communities, driving the focus of the 2020 conference on the converging crises of public health, economic insecurity, and social justice.

Michael Anne Kyle @michaelannica "Being in year 5 of a PhD in health policy, I have spent a very large amount of time thinking about waste, moral hazard, unnecessary utilization, consumer decision-making, etc. And yet this week I went to the ED for a broken toe."

The percentage of telehealth visits for mental health services rose in most regions this year, possibly due to increased stress, FAIR Health's president Robin Gelburd says. Variation could be due to differences in substance use disorder diagnoses.

A periodic feature delivering insights from health and tech leaders on how the pandemic is changing the medical system.

Its been less than a year since Chinese scientists shared the Covid-19 genetic sequence, allowing the sprint for a vaccine to commence. Now, states have until the end of this week to tell the federal government how they plan to distribute shots that could be shipped within 24 hours of FDA authorization.

Experts told us they have a multitude of concerns over how the vaccines are allocated to states, wholl get the shots first, and misinformation spreading online.

This is the first time in the history of our country where we are launching multiple novel vaccines all at once that have different dosing, complex scheduling and challenging transport, storage and administration requirements, says Vivian Riefberg, former co-lead of McKinseys U.S. health care practice and now professor of practice at University of Virginias Darden School of Business. Combine this with at least 50 different high level distribution plans (each state) using a range of inexperienced and experienced health care professionals in delivering these products. Then add to that overall market confusion for consumers about the various products. Will we have the right coordinated logistics and campaign to ensure the vast majority of people have timely access and take the vaccine?

David Blumenthal, president of the Commonwealth Fund, which has studied states immunization track records, said his big concern is that many states may not have the resources they need to implement an emergency population-wide immunization plan. Commonwealths research, he said, found huge variation across states in their past success in vaccinating their populations against influenza and other infectious diseases. States also display considerable differences in their ability to immunize populations of color, who suffer disproportionately from Covid-19.

We should leverage primary care providers, who have built trusting relationships with their patients, to educate the public and administer vaccines, says Farzad Mostashari, CEO of Aledade and a former national coordinator for health IT in the Obama administration. As front line workers, they should also be prioritized to receive the vaccines themselves. My biggest concern is that distribution may leave out smaller independent practices, especially in rural and underserved areas where their participation is needed most.

Im less concerned about the nuts and bolts of physical distribution, says Robert Wachter, professor and chair of the Department of Medicine at University of California, San Francisco. Im more concerned about misinformation about side effects ... When you vaccinate 200 million people, a fair number of them will have strokes, cancer or heart attacks in the months after vaccination. Some will spin these into vaccine side effects.

Paul Barrett, deputy director of the NYU Stern Center for Business and Human Rights, worries that the drumbeat of false information online will discourage vaccination. Even before Covid-19, a loud chorus of anti-vaxxer activists, some of whom wrapped their views in various conspiracy theories, had become prominent on the major social media platforms, he said. The coming Covid-vaccination push will almost certainly provoke this contingent into redoubling their activities.

GOOGLES LATEST BID FOR HOSPITAL AND PATIENT DATA: The tech giants forays into health care havent always been welcomed: Its data-collection partnership with the hospital chain Ascension is still under HHS investigation for potential violations of the health privacy law HIPAA. But POLITICO's Mohana Ravindranath writes Googles cloud business is now marketing itself as a technical facilitator for hospitals and insurers, as part of the broader push to share patient data between electronic health records and apps.

Google executives say the effort can relieve hospitals and insurers from the burden of doing a huge technical undertaking on their own. Beyond that, the company is prodding the industry to think bigger about how the digital overhaul can improve patient care, said Google Clouds global health care director Aashima Gupta. This could include new ways to analyze clinical data or building apps that could help patients manage their condition.

Patients may want better access to their health data, but its not clear if theyll trust this latest venture to safeguard theirs. Asked whether shes seen pushback from providers wary of Silicon Valleys expansion into health care, Gupta stressed the company aimed to be sensitive to the needs of clinicians and patients alike. "Consumers are demanding better ways to access their data," she said.

THE PROMISE AND PITFALLS OF AI: Artificial intelligence has been eyed as an easy fix for a health care system facing strain from the pandemic, an aging population and rising costs. Still, the technology raises ethical, legal, economic, and social questions policymakers must confront in order to realize its full potential, concludes a new report to Congress.

Jacquelyn Martin/AP Photo

The Government Accountability Office and the National Academy of Medicine in a joint inquiry acknowledge that AI has shown promise in predicting health outcomes, recommending treatment and helping to record clinical notes, among other tasks. But significant challenges persist. For instance, the technical experts building these tools cant always rely on high-quality data, potentially perpetuating bias that affects patient care. And how these systems work arent always transparent to patients or clinicians, which can undermine trust.

Given those trade-offs, the report offers six paths for Congress and federal regulators to consider, though it offers no recommendations. These include allowing the technology to flourish without government intervention (though GAO noted this could also increase disparities) and working with outside groups to encourage better data sharing and AI testing. Authors noted that groups with proprietary data and technology might be reluctant to participate.

TRUMP ADMIN CEMENTS VIRTUAL CARE CHANGES: The federal Medicare agency on Tuesday approved expanded coverage of telehealth services, ensuring some pandemic policies will last beyond the public health emergency period. Medicare will pay for 60 more telehealth services, though the changes wont do much for patients outside rural areas when the emergency declaration is lifted. Medicare payment law generally restricts telehealth coverage to patients in rural areas, unless Congress makes a change.

Alcoholics Anonymous goes to Zoom, reports the New York Times.

Science Magazine probes HHSs controversial coronavirus data system, alleges discrepancies.

STAT delves into a new artificial intelligence feat from the folks at Google DeepMind: folding proteins.

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Mini kidneys bioprinted in the lab – Lab + Life Scientist

December 4th, 2020 12:25 am

Researchers from the Murdoch Childrens Research Institute (MCRI) and biotech company Organovo have used cutting-edge technology to bioprint miniature human kidneys in the lab, paving the way for new treatments for kidney failure and possibly lab-grown transplants. Their study has been published in the journal Nature Materials.

Like squeezing toothpaste out of a tube, extrusion-based 3D bioprinting uses a bioink made from a stem cell paste, squeezed out through a computer-guided pipette to create artificial living tissue in a dish. According to MCRI Professor Melissa Little, a world leader in modelling the human kidney, this new bioprinting method is faster and more reliable than previous methods, allowing the whole process to be scaled up. 3D bioprinting could now create about 200 mini kidneys in 10 minutes without compromising quality.

From larger than a grain of rice to the size of a fingernail, bioprinted mini kidneys fully resemble a regular-sized kidney, including the tiny tubes and blood vessels that form the organs filtering structures called nephrons. Prof Little said the mini organs will be used to screen drugs to find new treatments for kidney disease or to test if a new drug was likely to injure the kidney.

Drug-induced injury to the kidney is a major side effect and difficult to predict using animal studies; bioprinting human kidneys are a practical approach to testing for toxicity before use, she said.

In the study, researchers tested the toxicity of aminoglycosides a class of antibiotics that commonly damage the kidney. Prof Little said, We found increased death of particular types of cells in the kidneys treated with aminoglycosides.

By generating stem cells from a patient with a genetic kidney disease, and then growing mini kidneys from them, also paves the way for tailoring treatment plans specific to each patient, which could be extended to a range of kidney diseases.

Prof Little said the study also showed that 3D bioprinting of stem cells can produce large enough sheets of kidney tissue needed for transplants. She noted, 3D bioprinting can generate larger amounts of kidney tissue but with precise manipulation of biophysical properties, including cell number and conformation, improving the outcome.

Prof Little said prior to this study the possibility of using mini kidneys to generate transplantable tissue was too far away to contemplate, but that may no longer be the case.

The pathway to renal replacement therapy using stem cell-derived kidney tissue will need a massive increase in the number of nephron structures present in the tissue to be transplanted, she said.

By using extrusion bioprinting, we improved the final nephron count, which will ultimately determine whether we can transplant these tissues into people.

Image credit: stock.adobe.com/au/bluebay2014

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Telix Pharmaceuticals Limited Acquires TheraPharm GmbH, Broadening Reach to Hematologic Cancers and Transplant Medicine – BioSpace

December 4th, 2020 12:25 am

MELBOURNE, Australia and BAAR, Switzerland, Nov. 29, 2020 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) announces it has entered into an agreement with Scintec Diagnostics GmbH (Scintec) to acquire TheraPharm GmbH (TheraPharm), a Swiss-German biotechnology company developing innovative diagnostic and therapeutic solutions in the field of hematology.

The acquisition of TheraPharm provides Telix with access to a portfolio of patents, technologies, production systems, clinical data and know-how in relation to the use of Molecularly Targeted Radiation (MTR) in hematology and immunology. TheraPharm is developing antibody MTR technology against CD66, a cell surface target highly expressed by neutrophils (a type of white blood cell) and tumor-infiltrating lymphocytes. As such, the technology has potentially very broad applications in the diagnosis and treatment of hematologic diseases (e.g. blood cancers), lymphoproliferative disorders and immune-mediated diseases (e.g. lupus, and multiple sclerosis). Of particular interest is the demonstrated use of the technology to safely and effectively perform bone marrow conditioning (BMC) prior to bone marrow stem cell transplant.

Telix CEO, Dr. Christian Behrenbruch stated, Telix is committed to extending and improving the lives of patients with serious diseases. As such, the acquisition of TheraPharm and its MTR assets are uniquely aligned to Telixs mission and technical strengths in antibody engineering and radiochemistry. TheraPharms technology has a significant role to play in BMC and stem cell transplantation across a broad range of blood cancers and rare diseases. The current approach to BMC employs highly toxic drugs that have a poor morbidity and mortality profile, and for which many patients are ineligible. MTR offers an excellent safety profile that may greatly expand the number of patients able to undergo life prolonging stem cell transplantation while greatly reducing the hospitalisation burden and cost associated with such procedures.

TheraPharm co-founder and Managing Director, Dr. Klaus Bosslet added, Over the past 5 years, TheraPharm, in collaboration with Dr. Kim Orchard from the University of Southampton (UK), has made excellent progress developing 90Y-besilesomab for the treatment of hematologic cancers and several related conditions including multiple myeloma, leukemia and amyloidosis. This unique asset is a logical addition to Telixs portfolio, offering a potentially rapid development path to a first commercial indication for the treatment of patients with SALA, while at the same time having potentially broad applications for stem cell transplantation in patients with more common cancers of the blood, including multiple myeloma and leukemia. We look forward to joining the Telix team in order to expedite the development of products for this under-served field.

Full transaction details, including financial terms, can be found via the Telix website and ASX portal here.

About Hematopoietic Stem Cell Transplant (HSCT)

Bone marrow conditioning (BMC) followed by hematopoietic stem cell transplantation (HSCT) is presently performed to treat patients with hematologic malignancies (blood cancers), with the objective of extending patient survival or achieving cure. HSCT is also performed for a broad range of non-cancer conditions. HSCT is preferentially performed in countries of high income (Europe >30,000, Americas >20,000, worldwide >65,000 p.a., respectively) and is growing at around 5% annually.

About Systemic Amyloid Light-Chain Amyloidosis (SALA)

SALA is a rare, but serious protein deposition disease, caused by a protein known as amyloid that is produced by abnormal plasma cells residing in the bone marrow. As amyloid accumulates in the organs of the body, organ function will eventually deteriorate, ultimately causing organ failure. SALA has an estimated prevalence of 30,000 and 45,000 in United States and Europe, respectively and while a rare disease, SALA portends a very poor prognosis, with a median survival from diagnosis of ~11 months if untreated.

The current standard of care comprises of induction therapy (typically cyclophosphamide, bortezomib, dexamethasone) plus high dose melphalan BMC, followed by HSCT. This approach is typically only accessible to a small proportion of patients (<20%) who are able to tolerate induction therapy and melphalan BMC.

About Telix Pharmaceuticals Limited

Telix is a clinical-stage biopharmaceutical company focused on the development of diagnostic and therapeutic products using Molecularly Targeted Radiation (MTR). Telix is headquartered in Melbourne, Australia with international operations in Belgium, Japan and the United States. Telix is developing a portfolio of clinical-stage oncology products that address significant unmet medical needs in prostate, kidney and brain cancer. Telix is listed on the Australian Securities Exchange (ASX: TLX). For more information visit http://www.telixpharma.com.

AboutTheraPharm GmbH

TheraPharm is a biotechnology company specialised in the research, development and manufacturing of monoclonal antibodies for targeted radiation of hematopoietic malignant and non-malignant diseases, lymphoproliferative diseases, conditioning for allogeneic stem cells as well as in diagnostics of inflammatory diseases and bone marrow metastases.

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Government of Canada and JDRF Canada announce new research funding to accelerate stem cell-based therapies for type 1 diabetes – Philippine Canadian…

December 4th, 2020 12:25 am

There are more than 300,000 Canadians living with type 1 diabetes (T1D), an autoimmune disease with no known cause or cure, resulting in the dysfunction, damage or loss of pancreatic beta cells that produce insulin in our bodies. People with T1D must treat themselves with insulin several times per day to keep their blood glucose levels normal, and despite their best efforts, they often experience serious, and even life-threatening, complications.

To mark the end of Diabetes Awareness Month, Sonia Sidhu, Member of Parliament for Brampton South, on behalf of the Honourable Patty Hajdu, Minister of Health, announced an investment of $6 million through the CIHR-JDRF Partnership to Defeat Diabetes for two Canadian research teams to accelerate the development of stem cell-based therapies for the treatment of T1D.

Stem cells show great promise as a source of insulin-producing cells that could be transplanted to provide a new source of insulin, to replace dysfunctional, damagedor lost pancreatic beta cells. Canada has a remarkable legacy in leading discoveries in this area. Stem cells were discovered in Toronto in 1961, and in 2000, a team in Edmonton were the first to pioneer transplantation of pancreatic islets (the part of the pancreas that contains insulin-producing cells). These achievements represent important steps toward a treatment that will allow people with T1D to live healthy lives without daily insulin injections.

The research teams are led by Dr. Maria Cristina Nostro at the University Health Network and the University of Toronto and Dr. Francis Lynn at the BC Childrens Hospital Research Institute and the University of British Columbia. The teams will build on Canadas demonstrated research excellence and leadership in clinical islet transplantation, stem cell biology, diabetes, immunology and genetic engineering to accelerate stem cell-based therapies for T1D. They will work in collaboration with other Canadian researchers to tackle some of the biggest scientific challenges that impede our progress in this area and move us closer to a future where people with T1D will no longer rely on insulin therapy.

This funding was provided by the Canadian Institutes of Health Research Institute of Nutrition, Metabolism and Diabetes (CIHR-INMD), and JDRF Canada, through the CIHR-JDRF Partnership to Defeat Diabetes established in 2017. Each partner will invest $3 million over five years. This investment is part of a large research initiative,100 Years of Insulin: Accelerating Canadian Discoveries to Defeat Diabetes, funded by CIHR and partners. This initiative commemorates the 100th anniversary of the discovery of insulin to be marked in 2021a discovery that changed the lives of millions of Canadians and people around the world and won researchers Sir Frederick Banting and John Macleod the Nobel Prize in Physiology or Medicine.

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News briefing: Four biotechs announce IPO terms, setting the pace to round out a busy year; FDA sets PDUFA date for Kadmon’s graft-versus-host drug -…

December 4th, 2020 12:25 am

Four more biotechs set the terms for their IPOs, lining up yet another busy week on Wall Street.

Silverback Therapeutics, which initially filed for a $100 million raise, is now shooting for $125 million from 7 million shares at a range of $17 to $19. About $70 million is tagged for the companys lead Phase I/Ib antibody-drug conjugate, SBT6050, for advanced or metastatic HER2-expressing solid tumors. Interim data from the Phase I dose-escalation cohorts are expected in the second half of 2021. Another $55 million is set aside for Silverbacks two other candidates, which have yet to reach the clinic.

Kinnate Biopharma is looking for a $170 million raise, and set a $16 to $18 range for its 10 million share offering. About $105 is earmarked for its RAF inhibitors, including its lead preclinical candidate KIN002787 for patients with lung cancer, melanoma and other solid tumors. An IND is coming in the first half of 2021, the company says. Back in August, it hooked a $98 million Series C.

Seer also set its shares at a $16 to $18 range. The company, which is working on next-gen proteome analysis tests, is offering 8.8 million shares, going for a $150 raise. About $65.0 million would go to its Proteograph Product Suite, which already has one collaborator and could have a second by the end of the year.

Sigilon Therapeutics is seeking $101 million from 5.6 million shares at a $17 to $19 range. Between $30 to $35 million will be set aside for its lead candidate, SIG-001, which is in a Phase I/II trial to prevent bleeding episodes in patients with hemophilia A. Another $30 to $35 million would fund a scale-up of the companys GMP manufacturing processes SIG-001 and SIG-005, its preclinical candidate for patients with mucopolysaccharidosis type 1, or MPS-1.

More than 72 biotech and biopharma companies and counting have hit Nasdaq so far this year, and head of healthcare listings Jordan Saxe predicted rounding out the year with just under $14 billion in proceeds.

Kadmons chronic graft-versus-host drug belumosudil is in the FDAs hands.

The agency accepted the biotechs NDA under its real-time oncology review (RTOR) pilot program, and set the PDUFA date for May 30, 2021. The submission was based on positive results from a pivotal, open-label trial dubbed ROCKstar, which enrolled 132 patients who had received at least two prior lines of therapy.

Belumosudil is designed to tamp down the inflammatory response seen after hematopoietic stem cell transplant by blocking Rho-associated coiled-coil kinase 2 (ROCK2). Instead of a comparator arm, Kadmon set the bar at a 30% overall response rate, based on conversations with the FDA. Patients were given 200 mg of the drug either once or twice daily. At six months after the completion of enrollment, Kadmon saw an ORR of 73% and 75% in the respective arms.

Thats it for data, until the full report is read out at ASH in December. The companys stock $KDMN was up 6.68% on Monday, at $4.39 per share.Kadmon was founded by Sam Waksal, the biotech exec who was sentenced to prison for his insider trading conviction involving Martha Stewart.

Hookipa Pharma said no one from a small group of Phase II participants treated with its prophylactic cytomegalovirus vaccine came down with the disease, according to interim results.

The analysis was based on 41 participants 8 of whom received 3 doses, 19 of whom received 2 doses, and 14 of whom received a placebo. Compared to the placebo, researchers saw a 48% reduction in CMV viremia, and a 42% reduction in the use of antiviral therapy. There were no cases of CMV disease in the treatment arm, compared to 2 in the placebo group.

While these interim data are from a small group of patients, they offer early insight into the potential of a three-dose schedule of HB-101 to help protect kidney transplant recipients against CMV disease, CEO Joern Aldag said in a statement.

The candidate is going head-to-head with Modernas mRNA-1647, which produced positive results from a proof-of-concept study back in January. Researchers said they saw an increasing level of neutralizing antibody titers in those who had received a third vaccination.

This could really be a company builder, CEO Stphane Bancel said at the time, adding that the candidate could turn into a $2 billion to $5 billion annual franchise.

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Global Regenerative Medicine Market to Rise Impressively at 26.1% CAGR and Hit USD 151,949.5 Million by 2026; Integra’s Latest Product Offerings to…

December 4th, 2020 12:24 am

Pune, India, Dec. 01, 2020 (GLOBE NEWSWIRE) -- The global regenerative medicine market size is likely to expand considerably in the coming years due to growing applications in the treatment of chronic diseases. The market was valued at US$ 23,841.5 Million in 2018. Fortune Business Insights states that the market will reach US$ 151,949.5 Million by the end of 2026, thereby exhibiting a CAGR of 26.1%. North America generated maximum revenue of US$ 9,128.2 Million in 2018 and is expected to dominate the global regenerative medicine market throughout the forecast period. Due to presence of substantial number of key market players based in U.S., presence of research institutes involved in development of novel therapeutics and availability of advanced technologies are attributive to the high number of clinical trials in North America.

Key Industry Developments:

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Increasing Investment in R&D of Regenerative Medicine to Enable Growth

The growing investment in research and development of regenerative medicine has made a significant contribution to the growth of the global market. The increasing investment from private as well as government organizations has had a positive impact on the global market. IN March 2018, SanBio collaborated with Hitachi Chemical Advanced Therapeutics Solutions for developing regenerative medicine across a wide range of applications.

Novartis Receives EU Approval for Luxturna

The severity of chronic diseases has led to the demand for efficient medicines. The ability of regenerative medicine to treat severe life-threatening diseases in an efficient manner has created a huge demand for the products across the world. Increasing drug approvals have contributed to the rising uptake for regenerative medicines.

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In 2018, Novartis received usage approval from the European Union for its latest regenerative medicine Luxturna. The drug was used to treat and restore sight for people with vision impairment. Luxturna was widely useful in treatment of rare retinal diseases.

Integra LifeSciences Latest Product Offering Will Favor Market Growth

The advancements in regenerative medicine have fueled their demand across the world. Increasing product launches have contributed to the rising uptake of regenerative medicine across the world. In 2017, Integra LifeSciences announced the launch of Integra Dermal Regeneration Template Single Layer Thin.

Fortune Business Insights some of the leading companies that have made significant growth contributions to the global market. Besides this, the report identifies some of the attractive business strategies that have been adopted by renowned companies in the world.

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List of the leading companies that are operating in the Regenerative Medicine Market:

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Global Regenerative Medicine Market Segmentation:

By Product

Cell Therapy

Gene Therapy

Tissue Engineering

Platelet Rich Plasma

By Application

Orthopedics

Wound Care

Oncology

Others

By Distribution Channel

Hospitals

Clinics

Others

By Geography

North America (USA and Canada)

Europe (UK, Germany, France, Italy, Spain, Scandinavia and Rest of Europe)

Asia Pacific (Japan, China, India, Australia, Southeast Asia and Rest of Asia Pacific)

Latin America (Brazil, Mexico and Rest of Latin America)

Middle East & Africa (South Africa, GCC and Rest of Middle East & Africa)

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SECONDARY DATA SOURCES THAT WE REFER TO:

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Global Regenerative Medicine Market to Rise Impressively at 26.1% CAGR and Hit USD 151,949.5 Million by 2026; Integra's Latest Product Offerings to...

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Development of New Stem Cell Type May Lead to Advances In Regenerative Medicine – Newswise

December 4th, 2020 12:24 am

Newswise DALLAS Dec. 3, 2020 A team led by UT Southwestern has derived a new intermediate embryonic stem cell type from multiple species that can contribute to chimeras and create precursors to sperm and eggs in a culture dish.

The findings, published online this week in Cell Stem Cell, could lead to a host of advances in basic biology, regenerative medicine, and reproductive technology.

Cells in early embryos have a range of distinct pluripotency programs, all of which endow the cells to create various tissue types in the body, explains study leader Jun Wu, Ph.D., assistant professor of molecular biology. A wealth of previous research has focused on developing and characterizing nave embryonic stem cells (those about four days post-fertilization in mice) and primed epiblast stem cells (about seven days post-fertilization in mice, shortly after the embryo implants into the uterus).

However, says Wu, theres been little progress in deriving and characterizing pluripotent stem cells (PSCs) that exist between these two stages largely because researchers have not been able to develop a paradigm for maintaining cells in this intermediate state. Cells in this state have been thought to possess unique properties: the ability to contribute to intraspecies chimeras (organisms that contain a mix of cells from different individuals of the same species) or interspecies chimeras (organisms that contain a mix of cells from different species) and the ability to differentiate into primordial germ cells in culture, the precursors to sperm and eggs.

For this study, the researchers successfully created intermediate PSCs, which they named XPSCs from mice, horses, and humans.

Wu says that these results could eventually lead to an array of advances in both basic and applied research. For example, looking at gene activity in XPSCs from different species and interspecies chimeras could help researchers understand which signatures have been conserved through evolution. Examining the communication between cells in chimeras may help scientists identify strategies that could be used to accelerate the development of tissues and organs from stem cells used for transplantation. And using chimera-derived primordial germ cells to create sperm and eggs could aid in preserving endangered animal species and advancing infertility treatments.

These XPSCs have enormous potential. Our study helps open the door to each of these possibilities, says Wu, who is a Virginia Murchison Linthicum Scholar in Medical Research.

Wu notes that developing XPSCs presented a special challenge because the conditions that keep nave PSCs in a stable state are exactly the opposite from those that stabilize primed PSCs. While culture conditions for nave PSCs must activate a WNT cell-signaling pathway and suppress the FGF and TGF- pathways, the conditions to maintain primed PSCs must suppress WNT and activate FGF and TGF-.

Aiming for the preferred environment for XPSC derivation, Wu and his colleagues placed cells from early mouse embryos into cultures containing chemicals and growth factors that activate all three pathways. These lab-grown cells were extremely stable in culture and able to multiply without developing any further for approximately two years.

Additional experiments showed that these cells met the expectations researchers have long strived to meet of contributing to chimeras and directly differentiating into primordial germ cells. Wu and his colleagues made intraspecies chimeras of mice using cells derived from mice with different coat colors by injecting the cells into early mouse embryos. They also tracked the contributions of the XPSCs by tagging the cells with a fluorescent protein and then identifying them throughout the resulting offsprings body.

Wus team made interspecies chimeras by injecting horse XPSCs into early mouse embryos and allowing the embryos to develop in mice for several days. Surprisingly, although horses have a comparatively long gestational period nearly a year the researchers found that these foreign cells had contributed to mouse organ development, indicating that signals from the mouse cells determine organ developmental timelines.

Like XPSCs from other species, the human cells showed that they were capable of differentiating into a variety of tissues if culture conditions allowed them to progress in development, as well as directly form primordial germ cells in a dish.

Other UTSW researchers who contributed to this study include Leqian Yu, Yulei Wei, Carlos A. Pinzon Arteaga, Masahiro Sakurai, Daniel A. Schmitz, Canbin Zheng, and Emily D. Ballard. Yu and Wu are inventors on a patent application arising from this work.

This study was funded by the Cancer Prevention and Research Institute of Texas (CPRIT No. RR170076), the Hamon Center for Regenerative Science and Medicine, the Guangdong Provincial Key Laboratory of Genome Read and Write (No. 2017B030301011), and the Asahi Glass Foundation.

About UTSouthwestern Medical Center

UTSouthwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty has received six Nobel Prizes, and includes 23 members of the National Academy of Sciences, 17 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,500 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UTSouthwestern physicians provide care in about 80 specialties to more than 105,000 hospitalized patients, nearly 370,000 emergency room cases, and oversee approximately 3 million outpatient visits a year.

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Rare-disease foundation backs regenerative research – OutSourcing-Pharma.com

December 4th, 2020 12:24 am

CureDuchenne is a global nonprofit honed in on discovering a cure for Duchenne muscular dystrophy (DMD). Its funding arm, CureDuchenne Ventures, is investing in regenerative medicine firm Mesentech, as part of a joint funding collaboration with the Charles H Hood Foundation (which seeks to advance early-stage research for pediatric conditions).

CureDuchenne Ventures, the funding arm of CureDuchenne (a nonprofit aimed at discovering a cure for Duchenne muscular dystrophy) is investing in Mesentech Inc., a regenerative medicine company with a prodrug platform that selectively delivers therapeutics to bone. The project is part of a new joint funding collaboration with the Charles H. Hood Foundation (CHF), which seeks to advance early-stage research for pediatric conditions.

Lianna Orlando, CureDuchennes senior director of research, told Outsourcing-Pharma that bone fractures and quality of life are the top two main outcome measures identified by people with DMD.

Individuals with Duchenne are at a high risk for osteoporosis for multiple reasons that include reduced weight-bearing activity (which normally drives development of strong and dense bones), as well as a side-effect of glucocorticoid therapy, and from the effect of the chronic inflammatory response seen in dystrophin-deficient muscles, she said. Fractures in the vertebral bones of the spine are seen in up to 30% of boys with Duchenne, and in addition to being very painful may lead to spine deformity; fractures in the long bones in the arm or the leg are common, and in many cases lead to permanent loss of ambulation.

Whats more, she said, bone fractures can lead to additional complications.

Long bone fractures can release fat emboli into the bloodstream, which can obstruct blood flow in critical places, including the lungsmost often the lungs. Although rare, fat embolism syndrome is a particularly serious complication of bone fractures in Duchenne; in the most severe cases, fat embolisms can lead to death, Orlando told OSP.

The CureDuchenne investment supports Mesentechs lead program, MES-1007, into clinical development and its evaluation in DMD. There are currently no approved therapies for bone wastage for individuals affected by DMD.

Debra Miller, founder and CEO of CureDuchenne, said the investment and collaboration hold the potential to increase the groups impact.

Addressing bone morbidities that contribute to the loss of ambulation could significantly add quality of life to everyone affected by Duchenne, said Miller.

In addition to providing Mesentech with funding, CureDuchenne intends to play an active role in advancing the prodrug technology platform for DMD. Additionally, CureDuchenne chief scientific officer Michael Kelly will join Mesentechs scientific advisory board.

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Hematologist/Stem Cell Biologist to Direct Hematology and Cellular Therapy at Cedars-Sinai – Newswise

December 4th, 2020 12:24 am

Newswise LOS ANGELES (Dec. 1, 2020) -- Internationally recognized hematologist John P. Chute, MD, has been selected to direct the Division of Hematology and Cellular Therapy in the Department of Medicine at Cedars-Sinai Cancer. The physician-scientist also will serve as director of the Center for Myelodysplastic Diseases Research and associate director of the Board of Governors Regenerative Medicine Institute in the Department of Biomedical Sciences. Chute assumed his new post Nov. 23.

The selection of Chute, following a national search, reflects the importance of his pioneering research in blood-forming stem cells called hematopoietic stem cells, which can self-renew and generate all cell types found in the blood and immune system. Over the past decade, Chute's lab has discovered several growth factors produced by the cells that line the walls of blood vessels; they play a critical role in blood-forming stem cell regeneration.

"Dr. Chute is an exceptional addition to our faculty," saidDanTheodorescu, MD, PhD, director ofCedars-Sinai Cancer. "His international reputation as a physician-scientist who has made major contributions to stem cell and hematopoietic cell biologywill greatly contribute to positioning the newly created Division of Hematology and Cell Therapy as one of the best in the nation, while providing Cedars-Sinai Cancer patients with exciting new options for the treatment of blood malignancies."

In addition to his hematopoietic stem cell research, Chute said he looks forward to expanding Cedars-Sinai's CAR T-cell research and therapy. He describes the immune-boosting therapeutic as "transformative" for patients with advanced non-Hodgkinlymphoma,childhood acute lymphoblastic leukemiaand potentially several additional blood cancers.

CAR T-cell therapy is a type of immunotherapy in which patients' own immune cells, called T cells, are collected from their blood, and then an artificial receptor chimeric antigen receptor, or CAR is added to the cells' surface. The receptor enables the modified cells to specifically eradicate cancer cells. The cells are infused back into a patient's body intravenously, where they multiply and attack tumor cells.

"CAR T therapy has become an important treatment option for so many patients with advanced cancer who had no options before," Chute said. "That's what makes CAR T therapy so exciting."

Chute joins Cedars-Sinai from the David Geffen School of Medicine at the UCLA, where he was a professor of Medicine and Radiation Oncology in the Division of Hematology/Oncology and an investigator in the Broad Stem Cell Research Center.

Chute earned his medical degree at Georgetown University. He completed his residency in internal medicine and fellowship in Hematology/Oncology at the National Naval Medical Center. He completed his research training at the National Cancer Institute and the Naval Medical Research Institute.

"I'm excited to join the Cedars-Sinai Cancer faculty because of the opportunity to collaborate with the world-class scientists and top-tier physicians at the cancer center," Chute said. "Cedars-Sinai has always been a leading medical center and is deeply committed to basic and translational research, while also growing the hematology and cellular therapy specialties. I'm eager to play a leading role in that growth."

Clickhereto read more from the Cedars-Sinai Newsroom.

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Hematologist/Stem Cell Biologist to Direct Hematology and Cellular Therapy at Cedars-Sinai - Newswise

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How biotech startup Seragen is reworking infertility treatments with regenerative therapies – YourStory

December 4th, 2020 12:24 am

BABIES!

Most people love babies and want a few at some point in their life. But for about 30 million infertile couples in India, alone, babies are an out-of-reach pipe dream.

When Vasanthi Palanivel, a biotech scientist with two decades of experience, saw her friend, Srinivas Chari and his wife undergoing issues with conceiving a baby - and the immense grief that failed treatments caused - she decided to do something about it.

Vasanthis work in the biotech space focused specifically on tissue engineering and stem cell-based treatments, and she decided to use that expertise to develop a regenerative medicine-based infertility treatment, which she claims is better than most traditional treatments available today.

Fertility treatments are expensive - around Rs 1 lakh to Rs 1.25 lakh, per cycle, according to price ranges outlined online by leading IVF treatment centres, have low success rates, and, more importantly, are not covered by insurance. Only a percent of the 30 million infertile couples in India seek IVF treatments, and most of them target very specific problems with synthetic solutions, instead of restoring the patients health, holistically.

She says plasma-based treatments offer breakthroughs where traditional medicine has limited scope. Regenerative treatments are also generally safer, fast-acting, and go beyond just treating the symptoms and problem areas - they help restore organs, tissues and muscles to their ideal state so that the body can function as its supposed to.

So far, the startup has raised angel funding from several individual investors, and is open to more investments. Vasanthi, whos also the startups Chief Scientific Officer, says the venture is profitable, and growing at 100 percent, year on year.

Seragen, founded in 2018, treats over 10 conditions related to male and female infertility, including repeated miscarriages, low testosterone, erectile dysfunction, hormonal imbalance and endometriosis, among others.

The startup gets most of its clients via doctor referrals. On a pre-determined date, the patients case history detailing their medical condition, and their blood sample is collected by Seragens laboratory. The scientists then concoct a personalised fertility medicine, specific to the patients condition, and then send it back to the doctor for administering.

Image credit: Shutterstock

The process takes around 45 minutes, and the medication kicks in roughly 48 hours later.

The cost of the treatment is less than 10 percent of current fertility treatments, she adds.

So far, Seragen has treated over 2,000 patients. Its most requested treatments are for ovarian failures and endometrium. Women over 35 form a large part of the startups patient base.

Currently, the firm is piloting an immunology-based protocol with Indira IVF to target miscarriages - and Vasanthi says she hopes to publish encouraging results soon. The company is also working on a treatment to help prevent the death of mothers after they give birth.

Present in several cities in India already, Seragen is aiming to strengthen its presence domestically, as well as internationally. The startup says it will start operating in South Asian countries like Sri Lanka and Seychelles soon.

The startup competes with generic infertility treatment providers, as well as some stem cell-based therapy providers.

The global regenerative market is expected to reach $17.9 billion by 2025, from $8.5 billion in 2020, and growing at a CAGR of 15.9 percent, a Markets and Markets research report showed. Chronic diseases, genetic disorders and cancers, as well as rising investments in regenerative medicine research is expected to drive most of the growth, the research said.

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Resolution Therapeutics Announces a 26.6m Series A Financing from Syncona Ltd – PharmiWeb.com

December 4th, 2020 12:24 am

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AVITA Therapeutics Appoints Kathy McGee as Chief Operating Officer – Yahoo Finance

December 4th, 2020 12:24 am

VALENCIA, Calif, and MELBOURNE, Australia, Dec. 01, 2020 (GLOBE NEWSWIRE) -- AVITA Therapeutics, Inc. (NASDAQ: RCEL, ASX:AVH), a regenerative medicine company that is developing and commercializing a technology platform that enables point-of-care autologous skin restoration for multiple unmet needs, announced today the appointment of Kathy McGee as Chief Operating Officer, effective December 1, 2020.

Kathys extensive healthcare experience and industry insight are a welcome addition to AVITAs leadership team, said Dr. Mike Perry, AVITA Therapeutics Chief Executive Officer. In particular her broad operational experience within Regenerative Medicine will be critically important as we seek to bring our pipeline products through development and into the markets.

Ms. McGee joins AVITA with over 25 years of biopharmaceutical and life sciences experience, most recently serving as President of CnA Consulting Group, which focuses on providing specialized consulting services to the life sciences industry. Prior to CnA Consulting, Ms. McGee was the Vice President of West Coast Operations at Shire Pharmaceuticals Regenerative Medicine Division, formerly Advanced BioHealing, where she was a part of the leadership team responsible for manufacturing operations, strategic planning, capital expansion, and real estate. At Advanced BioHealing, Ms. McGee served as the Senior Vice President of Operations and General Manager, with responsibility for the companys manufacturing operations in La Jolla, CA. She has also held senior Operations leadership roles at Smith and Nephew and Advanced Tissue Sciences. She earned her Bachelor of Science in chemistry and mathematics from University College Galway Ireland, and holds a Masters degree in Business and Management from Webster University.

Authorized for release by the Chief Executive Officer of AVITA Therapeutics, Inc.

ABOUT AVITA THERAPEUTICS, INC.AVITA Therapeutics is a regenerative medicine company with a technology platform positioned to address unmet medical needs in burns, chronic wounds, and aesthetics indications. AVITA Therapeutics patented and proprietary collection and application technology provides innovative treatment solutions derived from the regenerative properties of a patients own skin. The medical devices work by preparing a RES REGENERATIVE EPIDERMAL SUSPENSION, an autologous suspension comprised of the patients skin cells necessary to regenerate natural healthy epidermis. This autologous suspension is then sprayed onto the areas of the patient requiring treatment.

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AVITA Therapeutics first U.S. product, the RECELL System, was approved by the U.S. Food and Drug Administration (FDA) in September 2018. The RECELL System is indicated for use in the treatment of acute thermal burns in patients 18 years and older. The RECELL System is used to prepare Spray-On Skin Cells using a small amount of a patients own skin, providing a new way to treat severe burns, while significantly reducing the amount of donor skin required. The RECELL System is designed to be used at the point of care alone or in combination with autografts depending on the depth of the burn injury. Compelling data from randomized, controlled clinical trials conducted at major U.S. burn centers and real-world use in more than 8,000 patients globally, reinforce that the RECELL System is a significant advancement over the current standard of care for burn patients and offers benefits in clinical outcomes and cost savings. Healthcare professionals should read the INSTRUCTIONS FOR USE - RECELL Autologous Cell Harvesting Device (https://recellsystem.com/) for a full description of indications for use and important safety information including contraindications, warnings and precautions.

In international markets, our products are marketed under the RECELL System brand to promote skin healing in a wide range of applications including burns, chronic wounds and aesthetics. The RECELL System is TGA-registered in Australia and received CE-mark approval in Europe.To learn more, visit http://www.avitamedical.com.

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTSThis letter includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as anticipate, expect, intend, could, may, will, believe, estimate, look forward, forecast, goal, target, project, continue, outlook, guidance, future, other words of similar meaning and the use of future dates. Forward-looking statements in this letter include, but are not limited to, statements concerning, among other things, our ongoing clinical trials and product development activities, regulatory approval of our products, the potential for future growth in our business, and our ability to achieve our key strategic, operational and financial goal. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Each forward- looking statement contained in this letter is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others, the timing of regulatory approvals of our products; physician acceptance, endorsement, and use of our products; failure to achieve the anticipated benefits from approval of our products; the effect of regulatory actions; product liability claims; risks associated with international operations and expansion; and other business effects, including the effects of industry, economic or political conditions outside of the companys control. Investors should not place considerable reliance on the forward-looking statements contained in this letter. Investors are encouraged to read our publicly available filings for a discussion of these and other risks and uncertainties. The forward-looking statements in this letter speak only as of the date of this release, and we undertake no obligation to update or revise any of these statements.

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AVITA Therapeutics Appoints Kathy McGee as Chief Operating Officer - Yahoo Finance

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EVerZom Raises 1.1M to Industrialize Its Exosome Biomanufacturing Platform – Business Wire

December 4th, 2020 12:24 am

PARIS--(BUSINESS WIRE)--EVerZom, a biopharmaceutical company specialized in the bioproduction of exosomes, announced today that it has raised 1.1 million in funding from institutional and private investors to develop its exosome bioproduction platform. This funding will speed up the platform development and scale-up, with the objective to allow routine clinical grade production by 2022.

Towards the emergence of new therapeutics in regenerative medicine

Exosomes are biological nanoparticles released by cells as an intercellular communication system to transport biomolecules. They have the ability to deliver therapeutics or regenerate tissue in several pathologies, including osteoarthritis, heart failure, and liver and kidney diseases, conditions which impact more than 150 million patients worldwide. Exosomes are now considered one of the safest and most promising future regenerative therapy solutions. They are also easy to store and have a low immunogenic profile, thus reinforcing their potential.

Increasingly, academic and industrial players are working on the therapeutic potential of exosomes. The main obstacle to the translation of exosomes into clinical development is industrial manufacturing while maintaining robust quality and reproducibility.

EVerZom's proprietary innovation consists in applying turbulence stimulation on cells to trigger massive exosome release. This approach enables the production of ten times more exosome ten times more rapidly than classical methods. The technology is being developed and already implemented in GMP certified systems, simplifying the clinical transfer.

This technology and the know-how developed around exosomes allows EVerZom to offer a scalable and reproducible exosome production process with robust quality controls. Everzom's internal R&D is continuing its work on the link between the qualification of exosomes and their application potential. EVerZom's ambition is to become the European leader in the bioproduction of exosomes.

"We are delighted with this financing opportunity, which will enable us to industrialize our proprietary technology and meet the growing demand of the exosome market. Our ambition is to foster the emergence of new exosome therapeutic strategies and to make them accessible to the largest possible number of patients as quickly as possible" explains Jeanne Volatron, Co-founder and President of EVerZom. http://www.everzom.com

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EVerZom Raises 1.1M to Industrialize Its Exosome Biomanufacturing Platform - Business Wire

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Regenerative Medicine Market to Witness Steady Expansion During 2025 – Murphy’s Hockey Law

December 4th, 2020 12:24 am

Regenerative medicine is a part of translational research in the fields of molecular biology and tissue engineering. This type of medicine involves replacing and regenerating human cells, organs, and tissues with the help of specific processes. Doing this may involve a partial or complete reengineering of human cells so that they start to function normally.

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Regenerative medicine also involves the attempts to grow tissues and organs in a laboratory environment, wherein they can be put in a body that cannot heal a particular part. Such implants are mainly preferred to be derived from the patients own tissues and cells, particularly stem cells. Looking at the promising nature of stem cells to heal and regenerative various parts of the body, this field is certainly expected to see a bright future. Doing this can help avoid opting for organ donation, thus saving costs. Some healthcare centers might showcase a shortage of organ donations, and this is where tissues regenerated using patients own cells are highly helpful.

There are several source materials from which regeneration can be facilitated. Extracellular matrix materials are commonly used source substances all over the globe. They are mainly used for reconstructive surgery, chronic wound healing, and orthopedic surgeries. In recent times, these materials have also been used in heart surgeries, specifically aimed at repairing damaged portions.

Cells derived from the umbilical cord also have the potential to be used as source material for bringing about regeneration in a patient. A vast research has also been conducted in this context. Treatment of diabetes, organ failure, and other chronic diseases is highly possible by using cord blood cells. Apart from these cells, Whartons jelly and cord lining have also been shortlisted as possible sources for mesenchymal stem cells. Extensive research has conducted to study how these cells can be used to treat lung diseases, lung injury, leukemia, liver diseases, diabetes, and immunity-based disorders, among others.

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Global Regenerative Medicine Market: Overview

The global market for regenerative medicine market is expected to grow at a significant pace throughout the forecast period. The rising preference of patients for personalized medicines and the advancements in technology are estimated to accelerate the growth of the global regenerative medicine market in the next few years. As a result, this market is likely to witness a healthy growth and attract a large number of players in the next few years. The development of novel regenerative medicine is estimated to benefit the key players and supplement the markets growth in the near future.

Global Regenerative Medicine Market: Key Trends

The rising prevalence of chronic diseases and the rising focus on cell therapy products are the key factors that are estimated to fuel the growth of the global regenerative medicine market in the next few years. In addition, the increasing funding by government bodies and development of new and innovative products are anticipated to supplement the growth of the overall market in the next few years.

On the flip side, the ethical challenges in the stem cell research are likely to restrict the growth of the global regenerative medicine market throughout the forecast period. In addition, the stringent regulatory rules and regulations are predicted to impact the approvals of new products, thus hampering the growth of the overall market in the near future.

Global Regenerative Medicine Market: Market Potential

The growing demand for organ transplantation across the globe is anticipated to boost the demand for regenerative medicines in the next few years. In addition, the rapid growth in the geriatric population and the significant rise in the global healthcare expenditure is predicted to encourage the growth of the market. The presence of a strong pipeline is likely to contribute towards the markets growth in the near future.

Global Regenerative Medicine Market: Regional Outlook

In the past few years, North America led the global regenerative medicine market and is likely to remain in the topmost position throughout the forecast period. This region is expected to account for a massive share of the global market, owing to the rising prevalence of cancer, cardiac diseases, and autoimmunity. In addition, the rising demand for regenerative medicines from the U.S. and the rising government funding are some of the other key aspects that are likely to fuel the growth of the North America market in the near future.

Furthermore, Asia Pacific is expected to register a substantial growth rate in the next few years. The high growth of this region can be attributed to the availability of funding for research and the development of research centers. In addition, the increasing contribution from India, China, and Japan is likely to supplement the growth of the market in the near future.

Global Regenerative Medicine Market: Competitive Analysis

The global market for regenerative medicines is extremely fragmented and competitive in nature, thanks to the presence of a large number of players operating in it. In order to gain a competitive edge in the global market, the key players in the market are focusing on technological developments and research and development activities. In addition, the rising number of mergers and acquisitions and collaborations is likely to benefit the prominent players in the market and encourage the overall growth in the next few years.

Some of the key players operating in the regenerative medicine market across the globe are Vericel Corporation, Japan Tissue Engineering Co., Ltd., Stryker Corporation, Acelity L.P. Inc. (KCI Licensing), Organogenesis Inc., Medtronic PLC, Cook Biotech Incorporated, Osiris Therapeutics, Inc., Integra Lifesciences Corporation, and Nuvasive, Inc. A large number of players are anticipated to enter the global market throughout the forecast period.

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TMR Research is a premier provider of customized market research and consulting services to busi-ness entities keen on succeeding in todays supercharged economic climate. Armed with an experi-enced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Regenerative Medicine Market to Witness Steady Expansion During 2025 - Murphy's Hockey Law

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Regenerative Medicine Market Research Report 2020 Includes 8-Year Forecasts due to the Impact of COVID-19 Adroit Market Research – The…

December 4th, 2020 12:24 am

The Regenerative Medicine Market Industry report shields a thorough investigation towards the competitive profile encompassing the market stake along with company outline of the major contributors functioning in the global Regenerative Medicine market. The market report offers a comprehensive summarization of product description, product type, technological development as well manufacturing analysis including cost, income, and gross analysis. The market comprises of past data related to growth rate, market price, volume and futuristic analysis of the Regenerative Medicine market.

Moreover, it describes factors that are responsible for influencing for the growth of the Regenerative Medicine market, demand, and supply as well as the challenges and opportunities tackled by the competitive Regenerative Medicine market. In addition, the report holds important information for the Regenerative Medicine market players that enables them to understand the overall market scenario and expand their Regenerative Medicine Market business stats.

The report covers a number of the players in the Regenerative Medicine market, including:

Integra LifeSciences Corporation; MiMedx Group, Inc.; AstraZeneca; F. Hoffmann-La Roche Ltd; Merck & Co., Inc.; Pfizer Inc.; and Baxter.

Request sample copy of this report at: https://www.adroitmarketresearch.com/contacts/request-sample/52

The Regenerative Medicine market report delivers screen-shot of major competitors, market tendencies together with the forecast over the next five years, with anticipated growth rates and the major factors impacting and driving growth, the market statistics and Regenerative Medicine Market Industry information resulting from the various blending of primary and secondary sources.

Global Regenerative Medicine Market Report tracks the all the significant market occasions. Social occasion of data from different fields and through proper discoveries, the report has firmly anticipated development of the worldwide Regenerative Medicine market including regions and different section.

Key points considered in the Regenerative Medicine market report: company profile, production cost structure of market, sales and income analysis of Regenerative Medicine market, production scrutiny by geological region, Regenerative Medicine market strategies considering the major aspects related to restraint, opportunities, driving factors, challenges and possible analysis of new Regenerative Medicine market projects and their investment structure.

Read complete report @ https://www.adroitmarketresearch.com/industry-reports/regenerative-medicine-market

The Global Regenerative Medicine market focuses on the major factors mentioned below:

1.A comprehensive outline of the Regenerative Medicine market has been analyzed, which involves the valuation of the different organization in the global market.2.Developing trends in various Regenerative Medicine market segments and geological market.3.Substantial changes in market strategies and Regenerative Medicine market synopsis.4.Market methodologies and market stakes of major players in the Regenerative Medicine market.5.Existing and future dimensions of Regenerative Medicine market on the basis of both cost and volume point of view.6.Estimates of latest Regenerative Medicine Market Industry trends and advanced development.7.Major references for the new entrants for stability in the global and competitive market.

To describe and classify the market for Regenerative Medicine market

Delivers complete data associated with the major factors inducing growth of the global Regenerative Medicine market that includes drivers, restraints, opportunities, and trends. Studies and predicts the market volume and market stake of Regenerative Medicine market, related to volume and values. The Regenerative Medicine market report examines the market breakdown and anticipates the market volume related to volume and value, for geographical regions that include growth regions over the globe. Investigates competitive expansion, associated with product introduction, developmental stability and agreement & mergers occurring in the Regenerative Medicine market. Tactically portrays the competitive players functioning in the Regenerative Medicine market.

Delivers complete data associated with the major factors inducing growth of the global Regenerative Medicine market that includes drivers, restraints, opportunities, and trends. Studies and predicts the market volume and market stake of Regenerative Medicine market, related to volume and values. The Regenerative Medicine market report examines the market breakdown and anticipates the market volume related to volume and value, for geographical regions that include growth regions over the globe. Investigates competitive expansion, associated with product introduction, developmental stability and agreement & mergers occurring in the Regenerative Medicine market. Tactically portrays the competitive players functioning in the Regenerative Medicine market.

In this Regenerative Medicine market report study, scrutiny of dealers and distributors is specified along with contact information. Also, the Regenerative Medicine market report includes the manufacturing plants, Regenerative Medicine Market Industry details of imports and exports, demand and supply chain, their ability, worldwide productivity, and revenue. Lastly, it provides the data related to research findings, Regenerative Medicine Market data sources, conclusion, and appendix.

For Any Query on the Regenerative Medicine market: https://www.adroitmarketresearch.com/contacts/enquiry-before-buying/52

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LUCA Science Announces Joint Research With Division of Cardiology, Field of Internal Medicine, Nagoya University Graduate School of Medicine on the…

December 4th, 2020 12:24 am

TOKYO--(BUSINESS WIRE)--LUCA Science (Tokyo, Japan), a biotechnology company pioneering a novel class of mitochondrial therapeutics, announced today that it will collaborate with the Division of Cardiology, Field of Internal Medicine, Nagoya University Graduate School of Medicine to initiate a joint research on the development of mitochondrial therapy for cardiac ischemicreperfusion injury.

Background of joint research

Ischemic heart disease (myocardial infarction and angina) still remains the leading cause of death in the world despite advances in treatment and prevention *1. Ischemia, the cause of these disorders, is a condition in which blood flow is temporarily blocked, interrupting the oxygen supply to the myocardial tissue and severely impairing its function. Blood reperfusion is required to recover tissue function. However, reperfusion causes an increase in intracellular calcium and reactive oxygen species (free radicals), which induce intracellular mitochondrial damage, cardiomyocyte necrosis, and arrhythmias, among other disorders.

The Division of Cardiology, Field of Internal Medicine, Nagoya University Graduate School of Medicine is conducting basic and clinical research on regenerative medicine, severe heart failure, arrhythmia and ischemic heart disease, and has extensive experience in research on the diagnosis and treatment of various cardiovascular diseases such as ischemic heart disease, arrhythmia, valvular heart disease, cardiomyopathy, pulmonary hypertension and peripheral arterial disease.

LUCA Science is developing an innovative platform of highly functional mitochondrial therapy using proprietary technologies: LUCA Sciences mitochondria can be stored and delivered as a biopharmaceutical agent. The bioenergetics enhancement, tissue protection and cellular functional improvement from the mitochondria therapy is expected to reduce the size of myocardial infarction due to ischemia-reperfusion injury and provide therapeutic benefits for acute myocardial infarction.

Comment from Prof. Toyoaki Murohara at the Division of Cardiology, Field of Internal Medicine, Nagoya University Graduate School of Medicine.

Mitochondria are very important organelles that produce energy in the cell. Until now, most research has been conducted to improve mitochondrial function using drugs, but this is a completely new and innovative research project that involves the direct administration of purified mitochondria into the body for therapeutic applications. We are planning to work with LUCA Science to study the effects of mitochondrial therapy on myocardial damage.

About LUCA Science

LUCA Science is a preclinical stage biopharmaceutical company pioneering a new class of mitochondrial therapy to restore cellular bioenergetics in dysfunctional or damaged tissues and organs. Mitochondria are the power plants in our cells that produce energy for our bodies. LUCA Science has developed a novel method to isolate proprietary functional mitochondria which can then be stored and delivered as a biopharmaceutical agent. The advanced delivery system can be applied not just for mitochondria but also other compounds that can improve bioenergetics in specific cells or tissues.

LUCA Science recently completed $9.8 million Series A financing.

*1: World Health Organization (WHO) Global Health Estimates 2016: Deaths by Cause, Age, Sex, by Country and by Region, 2000-2016. Geneva, World Health Organization; 2018.https://www.who.int/news-room/fact-sheets/detail/the-top-10-causes-of-death

LUCA Science Inc.Name : LUCA Science Inc.Headquarters : 3-8-3, Nihonbashi Honcho, Chuo-ku, Tokyo, 103-0023, JapanCEO : Rick C. Tsai DMD, MDEstablished : December 25th 2018Employees : 12URL : https://luca-science.com Business : Research and development of mitochondrial biopharmaceuticals

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Regenerative Medicine Products Market Size, Revenue, Share, Scope, Growth Rate And Forecast To 2027 – The Haitian-Caribbean News Network

December 4th, 2020 12:24 am

New Jersey, United States: Market Research Intellect has added a new report to its huge database of research reports, entitled Regenerative Medicine Products Market Size and Forecast to 2027. The report offers a comprehensive assessment of the market including insights, historical data, facts, and industry-validated market data. It also covers the projections using appropriate approximations and methods.

Regenerative Medicine Products Market Overview

The Regenerative Medicine Products Market Report provides comprehensive data on market dynamics, market trends, product growth rate, and price. The Regenerative Medicine Products market report has various facts and statistics assuming the future predictions of the upcoming market participants. In addition, it offers business security taking into account sales, profit, market volume, demand and market supply ratio. The in-depth study provides vital information related to market growth, driving factors, major challenges, opportunities, and threats that will prove to be very helpful for market participants in making upcoming decisions.

Regenerative Medicine Products Market: Competitive Landscape

The Regenerative Medicine Products Market report consists of the Competitive Landscape section which provides a complete and in-depth analysis of current market trends, changing technologies, and enhancements that are of value to companies competing in the market. The report provides an overview of sales, demand, futuristic costs and data supply as well as a growth analysis in the forecast year. The key vendors in the market that are performing the analysis are also clearly presented in the report. Their development plans, their growth approaches, and their merger and acquisition plans are also identified. Information specific to a keyword in each of these regions is also provided. This report also discusses the submarkets of these regions and their growth prospects.

Prominent players operating in the market:

Regenerative Medicine Products Market Segmentation

The report contains the market size with 2019 as the base year and an annual forecast up to 2027 in terms of sales (in million USD). For the forecast period mentioned above, estimates for all segments including type and application have been presented on a regional basis. We implemented a combination of top-down and bottom-up approaches to market size and analyzed key regional markets, dynamics and trends for different applications.

Regenerative Medicine Products Market Segment by Type:

Regenerative Medicine Products Market Segment by Application:

Regenerative Medicine Products Market Regional overview:

In the report, experts analyze and forecast the Regenerative Medicine Products market on a global as well as regional level. Taking into account all aspects of the market in terms of regions, the focus of the report is on North America, Europe, Asia Pacific, the Middle East and Africa, and South America. The prevailing trends and various opportunities in these regions are studied that can convince the growth of the market in the forecast period 2020 to 2027.

Reasons to Buy the Regenerative Medicine Products Market Report:

Outlook analysis of the Regenerative Medicine Products market sector with current trends and SWOT analysis. This study evaluates the dynamics, competition, industrial strategies and strategies of the emerging countries. This report has a comprehensive guide that provides market insights and detailed data on each market segment Market growth factors and risks are presented. More precise information provision on the Regenerative Medicine Products market for different countries. Provide visions on factors influencing the growth of the market. Market segmentation analysis, including quantitative and qualitative research considering the impact of economic and non-economic aspects Comprehensive company profiles with product offerings, important financial information and the latest developments.

If you have any custom requirements, please let us know and we will offer you the customized report as per your requirements.

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Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage, and more. These reports deliver an in-depth study of the market with industry analysis, the market value for regions and countries, and trends that are pertinent to the industry.

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CRISPR Therapeutics and Vertex to Host Investor Webcast to Review Data Presented at the 62nd American Society of Hematology Annual (ASH) Meeting and…

December 4th, 2020 12:24 am

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 01, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the companies will host an investor webcast on December 9, 2020 at 8:00 a.m. ET to review clinical data presented during the Plenary Scientific Session at the annual ASH Meeting and Exposition from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with sickle cell disease and beta thalassemia. The presentation will include speakers from Vertex and CRISPR Therapeutics as well as Haydar Frangoul M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center, and a principal investigator in the CTX001 clinical studies.

The conference call will be webcast live and a link to the webcast can be accessed on the CRISPR Therapeutics website at https://crisprtx.gcs-web.com/events in the Investors section under Events and Presentations and on the Vertex website at http://www.vrtx.com in the "Investors" section. To access the call via phone, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the companies websites for approximately 30 days.

This meeting is not an official program of the ASH annual meeting.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

Story continues

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about plans to review data presented at the annual ASH Meeting and Exposition from two ongoing Phase 1/2 clinical trials of CTX001 in patients with sickle cell disease and beta thalassemia, as well as the anticipated speakers participating in the investor webcast. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential that preliminary data from any clinical trial not to be indicative of final trial results; the potential that clinical trial results may not support registration or further development; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR THERAPEUTICS word mark and design logo and CTX001 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the expectations and plans to review data presented at the annual ASH meeting and exposition from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with sickle cell disease and beta thalassemia, and the anticipated speakers participating in the investor webcast. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, the potential for data from a limited number of patients may not to be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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