StemCell Therapy

PLEASANTON, Calif., Sept. 15, 2020 (GLOBE NEWSWIRE) -- 10x Genomics (Nasdaq: TXG) today announced it has begun shipping its Chromium Single Cell Multiome ATAC + Gene Expression solution to customers, marking the first commercial release of a product capable of simultaneously profiling the epigenome and transcriptome from the same single cell. This multi-omic approach provides customers with the ability to link a cells epigenetic program to its transcriptional output, enabling a better understanding of cell functionality and bypassing the need to infer relationships through computer simulations.

This is one of our most ambitious undertakings at the company, said Ben Hindson, co-founder and Chief Scientific Officer of 10x Genomics. By introducing the first solution that captures ATAC and gene expression simultaneously, researchers can gain even more clarity by combining two already powerful methods to profile biological systems at single cell resolution simultaneously for the first time.

The new solution builds on an array of new products launched by the company this year for both its Chromium platform for single cell analysis as well as its Visium platform for spatial genomics. Early customers already working with Chromium Single Cell Multiome ATAC + Gene Expression include Stanford University School of Medicine, Icahn School of Medicine at Mt. Sinai and Spains Centro Nacional de Anlisis Genmico.

My lab is interested in understanding why some immune cell types fail to fight the cancer, said Dr. Ansuman Satpathy, Assistant Professor of Pathology, Stanford University School of Medicine. We plan to use 10x Genomics' new assay to understand the epigenetic and transcriptional regulation of immune cell dysfunction directly in patient samples, and to use this information to precisely engineer more effective immunotherapies in the future.

Until now, we have relied on computational prediction to match a cell's epigenome to a single-cell gene expression profile, said Dr. Holger Heyn, leader of the single cell genomics team at Spains Centro Nacional de Anlisis Genmico that is working on delineating the dynamics underlying B-cell differentiation and activation. 10x Genomics new multiome assay will allow us to directly measure what before could only be predicted, and offers a new gold standard that will confirm how accurate these predictions had been.

"With this new technology, we can better understand the mechanisms affected by the non-coding risk genetic variation across a wide range of neuropsychiatric diseases, including Alzheimers, Parkinsons, Schizophrenia, bipolar disorder and major depression, along with different severity of neuropathology and clinical symptomatology," added Dr. Panagiotis Roussos, Associate Professor of Genetics and Genomics Sciences, Icahn School of Medicine at Mount Sinai.

By using Chromium Single Cell Multiome ATAC + Gene Expression, researchers can:

Chromium Single Cell Multiome ATAC + Gene Expression is shipping to customers. To learn more, visit https://www.10xgenomics.com/products/single-cell-multiome-atac-plus-gene-expression.

About 10x Genomics10x Genomics is a life science technology company building products to interrogate, understand and master biology to advance human health. The companys integrated solutions include instruments, consumables and software for analyzing biological systems at a resolution and scale that matches the complexity of biology. 10x Genomics products have been adopted by researchers around the world including 97 of the top 100 global research institutions and 19 of the top 20 global pharmaceutical companies, and have been cited in over 1,500 research papers on discoveries ranging from oncology to immunology and neuroscience. The companys patent portfolio comprises more than 775 issued patents and patent applications.

Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements generally can be identified by the use of forward-looking terminology such as may, will, should, expect, plan, anticipate, could, intend, target, project contemplate, believe, estimate, predict, potential or continue or the negatives of these terms or variations of them or similar terminology. These forward-looking statements include statements regarding 10x Genomics, Inc.s partnership activities, which involve risks and uncertainties that could cause 10x Genomics, Inc.s actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on managements current expectations, forecasts, beliefs, assumptions and information currently available to management, and actual outcomes and results could differ materially from these statements due to a number of factors. These and additional risks and uncertainties that could affect 10x Genomics, Inc.s financial and operating results and cause actual results to differ materially from those indicated by the forward-looking statements made in this press release include those discussed under the captions "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" and elsewhere in the documents 10x Genomics, Inc. files with the Securities and Exchange Commission from time to time. The forward-looking statements in this press release are based on information available to 10x Genomics, Inc. as of the date hereof, and 10x Genomics, Inc. disclaims any obligation to update any forward-looking statements provided to reflect any change in its expectations or any change in events, conditions, or circumstances on which any such statement is based, except as required by law. These forward-looking statements should not be relied upon as representing 10x Genomics, Inc.s views as of any date subsequent to the date of this press release.

Disclosure Information10x Genomics uses filings with the Securities and Exchange Commission, its website (www.10xgenomics.com), press releases, public conference calls, public webcasts and its social media accounts as means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

ContactsMedia:media@10xgenomics.comInvestors:investors@10xgenomics.com

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10x Genomics First to Market With Product to Simultaneously Capture Epigenome and Transcriptome - GlobeNewswire

DENVER, Sept. 15, 2020 (GLOBE NEWSWIRE) -- Mydecine Innovations Group Inc. (CSE:MYCO) (OTC:MYCOF) (FSE:0NFA) ("Mydecine" or the "Company"), today announced the Company has formed a special committee to evaluate a number of options to increase shareholders value.

Topics that the special committee will discuss include, but limited to, a potential spin out of certain company assets. Currently, the company owns and operates a number of growing US assets in the THC-cannabis and hemp-CBD space, along with distribution, that are being evaluated and assessed as potential spin out options with respect to the Mydecine's non-fungi related assets. Current shareholders would receive automatic shares of the SpinCo.

We are constantly looking at ways to increase our shareholder value. Our company has grown at an incredible rate and is quickly establishing itself as a leader in the functional mushroom and psychedelic medicine space, said Mydecine Chief Executive Officer, Joshua Bartch. With that said, the company has a number of highly valuable assets that could potentially create larger shareholder value if they were spun out into a more focused stand-alone vehicle. We are currently evaluating a number of potential options and partners to accomplish this goal."

Further information will be provided as this opportunity develops.

About Mydecine Innovations Group Inc.Mydecine Innovations Group is a publicly traded life sciences parent company dedicated to the development and production of adaptive pathway medicine, natural health products and digital health solutions stemming from fungi. Mydecines experienced cross functional teams have the dynamic capabilities to oversee all areas of medicine development including synthesis, genetic research, import/export, delivery system development, clinical trial execution, through to product commercialization and distribution. By leveraging strategic partnerships with scientific, medical, military, and clinical organizations, Mydecine is positioned at the forefront of psychedelic medicine naturally derived from fungi, therapeutic solutions, and fungtional mushroom vitality products. Our portfolio of unified companies, including Mydecine Health Sciences, Mindleap Health, and NeuroPharm focus on providing innovative and effective options that can provide millions of people with a healthier quality of life.

For further information about Mydecine Innovations Group Inc., please visit the Companys profile on SEDAR at http://www.sedar.comor visit the Companys website at http://www.mydecine.com.

On behalf of the Board of Directors:Joshua Bartch, Chief Executive Officercontact@mydecineinc.com

Corp Communication:Charles Lee, Investor Relationscorp@mydecineinc.com+1 (250) 488-6728

Public Relations:Cynthia Salarizadeh, PRpr@mydecineinc.com

The Canadian Securities Exchange has neither approved nor disapproved the contents of this news release and accepts no responsibility for the adequacy or accuracy hereof. This news release contains forward-looking statements, which relate to future events or future performance and reflect managements current expectations and assumptions. Such forward-looking statements reflect managements current beliefs and are based on assumptions made by and information currently available to the Company. Readers are cautioned that these forward-looking statements are neither promises nor guarantees, and are subject to risks and uncertainties that may cause future results to differ materially from those expected including, without limitation, the availability and continuity of financing, the ability of the Company to adequately protect and enforce its intellectual property, the Company's ability to bring its products to commercial production, continued growth of the global adaptive pathway medicine, natural health products and digital health industries, and the risks presented by the highly regulated and competitive market concerning the development, production, sale and use of the Company's products. Although the Company has attempted to identify important factors that could cause actual results to differ materially from those contained in forward-looking information, there may be other factors that cause results not to be as anticipated, estimated or intended. There can be no assurance that such information will prove to be accurate, as actual results and future events could differ materially from those anticipated in such information. These forward-looking statements are made as of the date hereof and the Company does not assume any obligation to update or revise them to reflect new events or circumstances save as required under applicable securities legislation. This news release does not constitute an offer to sell securities and the Company is not soliciting an offer to buy securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction. This news release does not constitute an offer of securities for sale in the United States. These securities have not and will not be registered under United States Securities Act of 1933, as amended, or any state securities laws and may not be offered or sold in the United States or to a U.S. Person unless so registered, or an exemption from registration is relied upon.

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Mydecine Innovations Group, Inc. to Create Special Committee for the Spin Out of US Related Assets - GlobeNewswire

Research coming out of the University of Pennsylvania School of Medicine last month demonstrated how artificial intelligence (AI) can be utilized to fight against opioid abuse. It focused on a chatbot which sent reminders to patients who underwent surgery to fix major bone fractures.

The research was published in the Journal of Medical Internet Research.

Christopher Anthony, MD, is the studys lead author and the associate director of Hip Preservation at Penn Medicine. He is also an assistant professor of Orthopaedic Surgery.

We showed that opioid medication utilization could be decreased by more than a third in an at-risk patient population by delivering psychotherapy via a chatbot, he said. While it must be tested with future investigations, we believe our findings are likely transferable to other patient populations.

Opioids are an effective treatment for pain following a severe injury, such as a broken arm or leg, but the large prescription of the drugs can lead to addiction and dependence for many users. This is what has caused the major opioid epidemic throughout the United States.

The team of researchers believe that a patient-centered approach with the use of the AI chatbot can help reduce the number of opioids taken after such surgerys, which can be a tool used against the epidemic.

Those researchers also included Edward Octavio Rojas, MD, who is a resident in Orthopaedic Surgery at the University of Iowa Hospitals & Clinics. The co-authors included: Valerie Keffala, PhD; Natalie Ann Glass, PhD; Benjamin J. Miller, MD; Mathew Hogue, MD; Michael Wiley, MD; Matthew Karam, MD; John Lawrence Marsh, MD, and Apurva Shah, MD.

The research involved 76 patients who visited a Level 1 Trauma Center at the University of Iowa Hospitals & Clinics. They were there to receive treatment for fractures that required surgery, and those patients were separated into two groups. Both groups received the same prescription for opioids to treat pain, but only one of the groups received daily text messages from the automated chatbot.

The group that received text messages could expect two per day for a period of two weeks following their procedure. The automated chatbot relied on artificial intelligence to send the messages, which went out the day after surgery. The text messages were constructed in a way to help patients focus on coping better with the medication.

The text messages, which were created by a pain psychologist specialized in pain and commitment therapy (ACT), did not directly go against the use of the medication, but they attempted to help the patients think of something other than taking a pill.

The text messages could be broken down into six core principles, : Values, Acceptance, Present Moment Awareness, Self-As-Context, Committed Action, and Diffusion.

One message under the Acceptance principle was: feelings of pain and feelings about your experience of pain are normal after surgery. Acknowledge and accept these feelings as part of the recovery process. Remember how you feel now is temporary and your healing process will continue. Call to mind pleasant feelings or thoughts you experienced today.

The results showed that the patients who did not receive the automated messages took, on average, 41 opioid pills following the surgeries, while the group who did receive the messages averaged 26. The 37 percent difference was impressive, and those who received messages also reported less overall pain two weeks after the surgery.

The automated messages were not personalized for each individual, which demonstrates success without over-personalization.

A realistic goal for this type of work is to decrease opioid utilization to as few tablets as possible, with the ultimate goal to eliminate the need for opioid medication in the setting of fracture care, Anthony said.

The study received funding by a grant from the Orthopaedic Trauma Association.

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AI Used To Identify Gene Activation Sequences and Find Disease-Causing Genes - Unite.AI

MEDIA ADVISORY

FOR IMMEDIATE RELEASE: Nature Communications: Published Monday, September 14, 2020

Newswise Corresponding Author:Yasmin Hurd, PhD, Director of The Addiction Institute of Mount Sinai, Icahn School of Medicine at Mount Sinai, New York, and other coauthors.

Bottom Line:Herion-addicted individuals have alterations in the expression a gene called FYN - a gene known to regulate the production of Tau, a protein that is highly elevated and implicated in neurocognitive disorders like Alzheimers disease. The study emphasizes that opioid use can affect the brain in a way that might increase vulnerability of neural systems that trigger neurodegeneration later in life; however, since these changes are epigenetic (alterations in gene function that are influenced by environmental factors and not alterations of the DNA itself), they are reversible and medications that have already been developed to target FYN for neurodegenerative disorders may be studied as a novel treatment for opioid addiction.

Results:Interestingly, findings were consistent across human, animal and cell models. Through post-mortem analysis of the brains of human heroin users, the team found that, specifically in neurons, the most significantly impaired epigenetic region is related to a gene called FYN. Essentially, heroin opened up the DNA at the FYN gene, which encodes a protein called tyrosine kinase FYN, that is strongly linked to synaptic plasticity and which directly results in production of Tau. Too much Tau in the brain is associated with neurodegenerative diseases. They observed that expression and activity of tyrosine kinase FYN was also induced in rats trained to self-administer heroin and also in primary striatal neurons treated with chronic morphine in vitro. Additionally, they demonstrated that inhibition of the FYN kinase (either via pharmacological means or through genetic manipulation) reduces heroin-seeking and heroin-taking behaviors.

Why the Research Is Interesting:The findings will increase awareness about the potential impact of heroin to alter neural systems related to neurodegenerative disorders. The findings also identify FYN inhibitors as a novel therapeutic treatment for heroin use disorders.

Who: Human brains from a cohort of subjects who succumbed to heroin overdose and normal controls, translational animal model of rats trained to self-administer heroin, and primary striatal neurons treated with chronic morphine in vitro.

When: Adult animals were exposed to heroin and their brains studied.

What:They performed unbiased, cell-type-specific, genome-wide profiling of chromatin accessibility, providing insights into epigenetic regulation directly in the brains of heroin-addicted individuals. To assess the causal relationship between heroin use and FYN pathology, they studied the brains of rats trained to self-administer heroin and they hit primary striatal neurons with chronic morphine in petri dishes to examine the effect at the individual cellular level.

Study Conclusions:By scanning the entire genome of heroin users to identify whether disturbances in how genes are turned on or off exist, Mount Sinai researchers found that heroin opened up the DNA at the FYN gene. The FYN gene is known to regulate the production of Tau, a protein implicated in neurodegenerative disorder like Alzheimers disease, meaning that heroin may put users at an increased risk of neurodegenerative disease later in life. Importantly, these novel findings suggest that FYN inhibitors (which have already been developed and are being assessed for use in Alzheimers disease) may be promising therapeutic tools for heroin-use disorder.

Paper Title: Chromatin accessibility mapping of the striatum identifies tyrosine kinase FYN as a therapeutic target for heroin use disorder

Said Mount Sinai's Dr. Yasmin Hurd of the research: Drug overdoses due to opioid abuse remain at epidemic levels and continue to rise precipitously during the current pandemic, with novel treatments desperately needed. Direct molecular insights into the heroin-addicted human brain are critical to guide future therapies. Our new study findings clearly open up new lines of treatment opportunities for opioid use disorder, which could benefit and potentially save the lives of so many.

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People with Heroin Addiction Have Unique Molecular Alterations to The Brain That Resemble Brain Disturbances Seen in Neurodegenerative Disorders Like...

Image credit: NASA

Duke researchers team up with NASA to explore gene-environment interactions in astronauts

By Alexis Kessenich

Astronauts on long-duration spaceflights (LDSF) face a number of risks to their health some more obvious that others like during dynamic events such as launch and landing. But there are also lesser-known dangers, such as spaceflight-associated neuro-ocular syndrome (SANS), a spectrum of physiologic and pathologic neuro-ophthalmic changes that include swelling of the optic disc, nerve damage and vision impairment.

An astronauts susceptibility of developing SANS remains largely unknown, but a team of researchers in the Center for Applied Genomics and Precision Medicine (CAGPM) is on a mission to discover what causes the predisposition.

The Nutritional Biochemistry Laboratory at NASAs Johnson Space Center, led byScott M. Smith, completed preliminary studies and found metabolomics and geneticdifferencesin astronauts who developed SANS. This ledto a broader evaluation of genetics, so the team at CAGPM engaged to help.

Rachel MyersandRicardo Henaowill lead the studys data science efforts.

Were exploring over 80 genes associated with these metabolic pathways and around 500 different genetic variants within those genes, says Rachel Myers, lead analyst for the study. Our team will test each to see if one or groups of these variants are associated with SANS.

The study is comprised of three different cohorts: one pre- and post-spaceflight cohort and two cohorts mimicking SANS and spaceflight environments on Earth.

For the first cohort, data, such as eye measurements, were collected from astronauts before and after an LDSF. For the second cohort, data will be collected from patients at the Mayo Clinic with polycystic ovary syndrome, which shares some characteristics with SANS. The third cohort is a 30-day head-down tilt bedrest study, which mimics spaceflight environments and has been shown to inflict similar ocular changes.

Because the sample size is so small, and the number of astronauts available to participate is limited, the team will look at ways to combine different variants together and test association with the phenotypes provided by NASAs preliminary study to see if they can find what causes the predisposition.

No one has ever looked at the genetic aspect of SANS before. Its going to be really interesting to explore non-traditional approaches for genetic associations, adds Myers.

At the end of the study, the team hopes to have both an understanding of what the genetic landscape of SANS is and a sense of what approaches are going to work for further investigation.

With a small cohort, we run the risk of finding something thats completely random, says Myers, so well do additional validation after our initial findings before making recommendations.

Ultimately, were exploring gene-environment interactions, addsGeoff Ginsburg, principal investigator on the study. The astronauts exposures in space from ionizing radiation and microgravity to extreme social isolation presents an exciting scientific opportunity to understand how this intense and hostile environment interacts with our genomes.

Myers says after the study the team also hopes to have a new pipeline in the Center for processing sequencing data to get genetic variants, which will help with future studies.

A solution for these astronauts is hopefully on the horizon. But, for now, the project is one small leap for CAGPM, one giant leap for genetic research!

Association of Genetics and B Vitamin Status With the Magnitude of Optic Disc Edema During 30-Day Strict Head-Down Tilt Bed RestAstronaut ophthalmic syndromeGenotype, Bvitamin status, and androgens affect spaceflightinduced ophthalmic changesSpaceflight-related ocular changes:the potential role of genetics, and the potential ofB vitaminsas a countermeasure

Originally posted here:
Houston, We Have an Eye Problem - Duke Today

SAN FRANCISCO, CA / ACCESSWIRE / August 24, 2020 / Tribe Public announced today that Dietrich Stephan, Chief Executive Officer of NeuBase Therapeutics, Inc. (NASDAQ:NBSE), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies using its scalable PATrOL platform to address genetic diseases, will present at Tribe Public's Presentation and Q&A Webinar Event at 8 am pacific/11 am eastern on Wednesday, August 26th, 2020. During this complimentary, 30-minute event, Dr. Stephan will introduce the NeuBase's next-generation gene silencing technology and discuss the company's progress with treatment candidates in Huntington's Disease (HD) and Myotonic Dystrophy (DM1). A question and answer session will follow the presentation. To register to join the complimentary event, please visit the Tribe Public LLC website: http://www.tribepublic.com, or send a message to Tribe's management at research@tribepublic.com to request your seat for this limited capacity Zoom-based event.

Dietrich A. Stephan, Ph.D. is an industry veteran who is considered one of the fathers of the field of precision medicine, having trained with the leadership of the Human Genome Project at the NIH and then going on to lead discovery research at the Translational Genomics Research Institute and serve as professor and chairman of the Department of Human Genetics at the University of Pittsburgh. Dr. Stephan has identified the molecular basis of dozens of genetic diseases and published extensively in journals such as Science, the New England Journal of Medicine, Nature Genetics, PNAS, and Cell. In parallel, Dr. Stephan has founded or co-founded more than ten biotechnology companies and has advised numerous other companies. These companies are backed by top-tier investors such as Sequoia Capital, KPCB, Thiel Capital, and Khosla Ventures as well as corporate partners such as Life Technologies, Pfizer, and Mayo Clinic. Notably, Dr. Stephan founded NeuBase Therapeutics in August 2018, took it public in 2019, and has since grown the company to market capitalization to the tune of hundreds of millions of dollars. Dr. Stephan received his Ph.D. from the University of Pittsburgh and his B.S. from Carnegie Mellon University.

ABOUT TRIBE PUBLIC LLCTribe Public LLC is a San Francisco, CA-based organization that hosts complimentary worldwide webinar & meeting events in the U.S. Tribe's events focus on issues that the Tribe members care about with an emphasis on hosting management teams from publicly traded companies from all sectors & financial organizations that are seeking to increase awareness of their products, progress, and plans. Tribe members primarily include Institutions, Family Offices, Portfolio Managers, Registered Investment Advisors, & Accredited Investors. Website: http://www.tribepublic.com.

ABOUT NEUBASE THERAPEUTICSNeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders. NeuBase is continuing its progress towards developing treatment candidates in Huntington's Disease (HD) and Myotonic Dystrophy (DM1.)

CONTACT:

Tribe Public, LLC.John F. Heerdink, Jr.Managing Partnerjohn@tribepublic.com

SOURCE: NeuBase Therapeutics, Inc.

View source version on accesswire.com:https://www.accesswire.com/603092/NeuBase-Therapeutics-CEO-Dietrich-A-Stephan-PhD-to-Present-at-Tribe-Publics-Presentation-and-QA-Webinar-Event-on-August-26-2020

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NeuBase Therapeutic's CEO, Dietrich A. Stephan, Ph.D., to Present at Tribe Public's Presentation and Q&A Webinar Event on August 26, 2020 - BioSpace

A woman spits into a tube so that her saliva can be tested for the presence of the novel coronavirus.

By Robert F. ServiceAug. 24, 2020 , 5:00 PM

Sciences COVID-19 reporting is supported by the Pulitzer Center and the Heising-Simons Foundation.

First, a technician pushes a pencil-length swab to the very back of your nasal passages. Then you pay $100 or more, and wait days for an answer. But faster, cheaper, more pleasant ways to test for the novel coronavirus are coming online. This month, the U.S. Food and Drug Administration granted emergency use authorization for two tests that sample saliva instead of nasal fluid, and more innovations are likely after FDA relaxed rules to allow new tests to be adopted more quickly. One candidate was announced last week: an experimental test, potentially faster and cheaper, that analyzes saliva in a new way.

There is real promise here, says Anne Wyllie, a microbiologist at Yale University who helped develop one of the new tests authorized this month. Takanori Teshima, chief of laboratory medicine at Hokkaido University, who also reported successful results testing saliva, agrees. It will have a big impact worldwide.

When SARS-CoV-2, the respiratory virus that causes COVID-19, emerged in December 2019, researchers scrambled to develop tests to detect the virus. Initially, they turned to a long-trusted technique for diagnosing respiratory infections: looking for viral genetic material in mucosal fluid, thought to be the best hunting ground for a respiratory virus, collected from deep in a patients nasal passages. Thats where the 15-centimeter swab comes in. The swab goes into a plastic tube with a chemical mixture that stabilizes the virus during transport to a diagnostics lab. There, technicians extract its genetic material and load it into a machine to carry out the polymerase chain reaction (PCR), which amplifies snippets of genetic material unique to the virus.

The procedure accurately identifies infections about 95% of the time. But the test is uncomfortable and, because collecting the swab requires close contact with patients, it puts medical personnel at risk of contracting the virus. Nobody wants to do that job, Teshima says.

Testing saliva for SARS-CoV-2 was no sure thing. Studies with other respiratory diseases showed saliva tests identified only about 90% of people for whom swab tests indicated an infection. But the appeal of an easier and safer test for the new coronavirus led researchers to try. People being tested simply drool into a bar-coded plastic tube, seal it, and drop it in a pouch thats shipped to a lab for PCR analysis. Because the procedure directly tests the fluid responsible for transmitting the virus between people, it may give a better indication of who is most contagious, says Paul Hergenrother, a chemist at the University of Illinois, Urbana-Champaign (UIUC), who led his universitys saliva test development.

As early as 12 February, researchers in Hong Kong and China reported inClinical Infectious Diseasesthat they couldidentify SARS-CoV-2 from salivain 11 of 12 patients whose swabs showed virus. Since then, groups in the United States, Singapore, and Japan have confirmed and further simplified the procedures, cutting out costly steps such as adding specialized reagents to stabilize the virus during transport and extract the genetic material.

In May, Wyllie and Yale colleagues teamed up with the National Basketball Association, which provided $500,000 to develop Yales saliva test; the test is now used for frequently testing players. On 4 August, the Yale team posted a preprint on medRxiv that said its saliva testagreed with swab results 94% of the time, at a cost of as little as $1.29 per sample, roughly 1/100 as much as commercial swab-based tests. On 15 August, FDA granted emergency approval for the SalivaDirect test, so that other FDA-approved labs can use the protocol. Last week, the agency extended approval to the UIUC test given its similarity to the Yale test. UIUC is now using its saliva test to test all 60,000 students, faculty, and staff twice a week, so they can isolate infected individuals as quickly as possible. Testing saliva makes sense scientifically, and it makes sense logistically, Hergenrother says.

Anew saliva test for RNA viruses, such as Zika and SARS-CoV-2, was reported last week inScience Advancesby researchers at the University at Albany. It could be even faster and cheaper because it does not need expensive lab equipment such as PCR machines. Rather than amplifying RNA to identify the virus, the approach uses snippets of DNA that bind to short, unique sections of RNA and change them from linear strands to loops. That alters how the RNA behaves in a common lab procedure known as gel electrophoresis, making it easy to detect. This is innovative, Wyllie says.

A relaxation of FDA rules announced last week could lead to still more variants. The new rules allow approved clinical labs to use tests they have developed without any additional approval step. In a tweet, Michael Mina, an epidemiologist at Harvard Universitys T.H. Chan School of Public Health, called FDAs decision Huge news!! because it would encourage labs to develop novel tests. It may also help speed development ofrapid tests that look for viral proteinsrather than genetic materialan efficient way to screen large numbers of asymptomatic people.

We dont need one test to be the end all and be all, Wyllie says. We just want options.

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New drool-based tests are replacing the dreaded coronavirus nasal swab - Science Magazine

A man in Hong Kong who recovered from COVID-19 was reinfected with the virus four-and-a half months later in the first recorded instance of a second infection, researchers at the University of Hong Kong said on Monday.

The findings suggest the disease can infect multiple times despite herd immunity.

According to the researchers, a genetic analysis showed that these two successive infections of the same patient had been caused by two different strains of the SARS-CoV-2 virus, responsible for COVID-19.

The 33-year-old man was first diagnosed with COVID-19 in late March but tested positive again while being tested after he returned to Hong Kong from Spain via Britain on August 15.

But he was found to be infected with a different coronavirus strain the second time around and was asymptomatic.

The two viral signatures were said to be "completely different", and belonged to different coronavirus lineages, or clades.

The first closely resembled strains collected in March and April, and the second strain matched the virus found in Europe -- where the patient had just been visiting -- in July and August.

"Our study proves that immunity for COVID infection is not lifelong -- in fact, reinfection can occur quite quickly," said Kelvin Kai-Wang To, a microbiologist at Hong Kong University's Faculty of Medicine and lead author of a forthcoming study that details the findings.

"COVID-19 patients should not assume after they recover that they won't get infected again," he told AFP.

To also said those who have overcome the virus should still practice social distancing, wear masks and practise hand washing.

Experts have voiced different opinions on how alarmed people should be at the new findings, which will be published in the peer-reviewed medical journal Clinical Infectious Diseases.

"This is a worrying finding for two reasons," said David Strain, a clinical senior lecturer at the University of Exeter Medical School.

"It suggests that previous infections are not protective. It also raises the possibility that vaccinations may not provide the hope that we have been waiting for."

If antibodies don't provide lasting protection, "we will need to revert to a strategy of viral near-elimination in order to return to a normal life", he said.

But others say the new case is likely to be rare. .

"It is to be expected that the virus will naturally mutate over time," said microbiologist Brendan Wren of the London School of Hygiene & Tropical Medicine.

"This is a very rare example of re-infection and it should not negate the global drive to develop COVID-19 vaccines."

The virus has killed over 800,000 people worldwide and there has been an uptick of new infections after many countries lifted lockdown measures.

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Hong Kong man becomes first patient reinfected with COVID-19, say researchers - Euronews

(Reuters) - The following is a roundup of some of the latest scientific studies on the novel coronavirus and efforts to find treatments and vaccines for COVID-19, the illness caused by the virus.

Hong Kong man has first documented COVID-19 reinfection

A 33-year-old man who had recovered from a severe case of COVID-19 in April was infected again four months later in the first documented instance of human reinfection, University of Hong Kong researchers said on Monday. In August, after returning from a trip to Europe, he was diagnosed again - but with a different strain of the virus. While the first infection landed him in the hospital, the second produced no symptoms. Genetically, the first virus was closely related to strains collected in March/April while the second was closely related to strains collected in July/August, the researchers wrote in a report seen by Reuters. "Our findings suggest that SARS-CoV-2 may persist in the global human population as is the case for other common-cold associated human coronaviruses," they said in a statement. "Since the immunity can be short lasting after natural infection, vaccination should also be considered for those with one episode of infection," researchers said. "Patients with previous COVID-19 infection should also comply with epidemiological control measures such as universal masking and social distancing," they added. The report has been accepted for publication in Clinical Infectious Diseases. (bit.ly/34v5Lii; reut.rs/3l823Rv)

Low oxygen in COVID-19 pneumonia linked to vascular widening

Widened small blood vessels in the lungs appear to be linked with the low oxygen levels seen in COVID-19 respiratory failure, a small study suggests. Researchers made the discovery by injecting saline with tiny microbubbles into the veins of 18 critically ill COVID-19 patients and tracked the bubbles using ultrasound. Normally, the bubbles would travel through the heart and enter the lungs, but would not get through the lungs because they would not fit through the capillaries. In more than 80% of these patients, however, the bubbles passed through the lungs and reached the blood vessels of the brain, which means the lung's capillaries were abnormally dilated, researchers reported earlier this month in the American Journal of Respiratory and Critical Care Medicine. The more bubbles that made their way beyond patients' lungs, the lower their oxygen levels, researchers said. This "may explain the disproportionate low oxygen levels seen in many patients with COVID-19 pneumonia," coauthor Dr. Hooman Poor of the Mount Sinai - National Jewish Health Respiratory Institute in New York City told Reuters. Researchers should consider testing drugs that would cause these patients' pulmonary blood vessels to constrict, he suggested. An editorial published in the journal on Friday points out that with dilated pulmonary blood vessels, a higher-than-usual volume of blood flows into the brain, which "raises the question of whether increased neurologic complications of COVID-19 could be related" to the dilation observed by the researchers. (bit.ly/32jSLsZ; bit.ly/3jeHtwL)

Genetic barcodes may help monitor coronavirus mutations

"Genetic barcodes" can help track how the new coronavirus spreads and mutates, researchers said on Saturday in the International Journal of Infectious Diseases. Based on the organization, or sequence, of the genetic code of the virus, the researchers identified 11 distinct SARS-CoV-2 "barcodes" that represent different clades, or lineages, descended from a common viral ancestor. "We were able to assign approximately 94% of the global sequenced genomes to one of the clades," Arnab Pain of King Abdullah University of Science and Technology in Saudi Arabia told Reuters. Different continents have different variations, his team found. The subtle differences in the genetic sequences represented by the barcodes may affect virus infectivity or illness severity, he noted. Most of the genetic profiles available for the study were from North America and Europe, with COVID-19 cases from other regions under-represented. The researchers plan to regularly update the barcodes. "This is a dynamic process, and some virus clades/subclades may eventually die-off in the future, and new clades may form," Pain said. "We will continue to monitor the viral mutations in the global scenario and share our observations with the scientific community." (bit.ly/31mZVNK)

Open tmsnrt.rs/3a5EyDh in an external browser for a Reuters graphic on vaccines and treatments in development.

Reporting by Nancy Lapid; Editing by Bill Berkrot

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First COVID-19 reinfection case reported; low oxygen levels linked to widening blood vessels in lungs - Reuters

By The Canadian Press on August 24, 2020.

CALGARY Alberta has become the third province in Canada to offer a promising treatment for individuals with specific types of leukemia and lymphoma.

The provincial government, in partnership with the Alberta Cancer Foundation, has earmarked $15 million for the program, commonly known as CAR T-cell therapy.

CAR T-cell therapy genetically reprograms a persons immune cells to attack cancer cells in the body and then after the genetic mutations happen in the laboratory infuse back into the patient, Alberta Health Minister Tyler Shandro said at a news conference in Calgary.

Its provided when conventional treatments are ineffective and the cancer reoccurs.

Alberta will be the third province to offer CAR T-cell therapy, which is also available in Ontario and Quebec.

Shandro said the therapy will be available to about 60 patients a year and the cost is about $400,000 per individual.

He said the funding will be used to conduct a clinical trial with CAR T-cells manufactured in Alberta at three sites: the Cross Cancer Institute, Tom Baker Cancer Clinic and Alberta Childrens Hospital.

The money will also pay for nursing staff, training and education for health-care workers, patient education and psychosocial support, lab and diagnostic imaging, and followup care.

Shandro said the results of the treatment have been positive.

CAR T-cell therapy trials have demonstrated durable remissions and potential cures in about 50 per cent of adults and 80 per cent of children and young adults. We want to provide Albertans with the same recovery opportunities, he said.

Treatment using cells manufactured in the U.S. is expected to begin by wintertime at the Tom Baker Cancer Centre, with the Alberta Childrens Hospital and Cross Cancer Institute to follow.

CAR T-cell therapy is a game-changing treatment that offers some patients their only chance to survive cancer, said Alicia Talarico, president of the Leukemia and Lymphoma Society of Canada.

Martha Kandt, from Lacombe, Alta., said she was given three months to live and sought the treatment in the U.S.

As a family, we decided the risk was worth it, to see if the treatment would work. I recently had a PET scan and I am in remission. Im so pleased this treatment is going to be available to Albertans who need it.

This report by The Canadian Press was first published on Aug. 24, 2020.

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Alberta the third province to offer promising leukemia and lymphoma treatment - Medicine Hat News

Prime editing is the gene-editing method that can insert, delete and do base swapping accurately. Prime editing also termed as genetic word processor precisely select the target DNA and replace genetic code. Targeting 75,000 different mutations and correcting 89% of genetic defects will drive the demand for prime editing. In 2017, the first gene editing in the human body was attempted. Gene editing in a patient with Hunters syndrome was tested for safety and concluded reliable shreds of evidence. Superior target flexibility and editing precision with minimal errors make Prime editing first preference over the other conventional technique such as CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). Application of prime editing in reversing Genetic disease will be a milestone in gene editing.

Increasing prevalence of genetic disease creates a huge opportunity for prime editing market. Successful preliminary results with a genetic disease like Tay Sachs and Sickle cell anaemia will drive the prime editing market. Technological advancements providing minimal error with this technique will fuel the growth of prime editing. Decreased cost of DNA sequencing will propel prime editing market for research and commercialization. Arising ethical and safety concerns will make prime editing highly regulated sector. This may limit the scope and can restraint the growing market. Detrimental effect on Genetic diversity due to genetic engineering in one way may limit the market scope.

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The global Prime Editing market is classified on the basis of application and end user:

Based on application, Prime Editing Market is segmented into following:

Based on end user, Prime Editing Market is segmented into following:

Prime Editing is the most recent invention has created a buzz in the market. Firms accessing conventional genome engineering technologies have rolled plans of transitioning to this new technology. The restructuring by the firms is either by building upon the technological capabilities or by merging or acquiring the firms which hold expertise in prime editing. Inscripta, one of the most innovative company has launched the worlds first benchtop platform for digital genome engineering. Inscriptas Onyx device that was launched in October 2019, will enable genome editing at an unprecedented scale and cheaper rate. In 2019, Beam Therapeutics collaborated with a premium start-up in prime editing segment Prime Medicine for Prime Editing Technology. Beam therapeutics holds expertise in precision genetic medicine using base editing technology. The market consolidation activities my giants depict that genome editing will be the largest revenue-generating segment for prime editing market.

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North America will drive the market for Prime Editing due to high prevalence of genetic disease and technological advancement in the U.S. and Canada. One in every 27 Jews, is carrying Tay Sachs disease gene. After North America, Europe is leading in patient pool for genetic diseases such Hemophilia and Cystic fibrosis. The genetic disease pool will drive the adoption for Prime editing treatments in this region. Asia-Pacific will remain at steady growth for Prime Editing market due less disease prevalence and focus on other therapies. Latin America and Middle East and Africa region will boost the market owing to the disease prevalence.

Examples of some of the market participants in Prime Editing market identified across the value chain Beam Therapeutics Inc., Precision BioSciences, Inscripta, Inc, Horizon Discovery Ltd., Sangamo Therapeutics, Inc., CRISPR Therapeutics., Intellia Therapeutics, Inc., and others

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Global Prime Editing Market to Witness Rapid Development During the Period 2020 2030 - Lake Shore Gazette

Governor Roy Cooper announced today that Beam Therapeutics (Nasdaq; BEAM), a biotechnology company developing precision medicines through DNA base editing, plans to build a manufacturing facility in North Carolinas Research Triangle Park, creating 201 jobs. Over a period of 5 years, the company expects to invest $83 million in the facility, which will support clinical and commercial manufacturing for the companys novel base editing programs.

"North Carolina is a leader in biotechnology, from the research in our labs to the states biomanufacturers, said Governor Cooper. Companies like Beam Therapeutics work in developing precision medicines will help keep North Carolina on the cutting edge of this industry.

Beam Therapeutics, with headquarters in Cambridge, Massachusetts, develops precision genetic medicines through base editing. The foundational level of genetic information is a single base letter in DNA, and an error to a single letter, known as a point mutation, can cause disease. Base editors have the ability to rewrite just a single letter, and thereby intervene at the most foundational level. Beams proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs.

We believe investment in strategic manufacturing capabilities is an important component of fully realizing the power of our base editing technology and achieving our vision to provide life-long cures to patients suffering from serious diseases, said John Evans, CEO of Beam Therapeutics. Research Triangle Park is a thriving biopharmaceutical hub, providing significant access to the broad range of talent we will need to make this vision a reality.

Although wages will vary depending on position, the average salary for the new positions will be $102,654. The average wage in Durham County is $71,756. The state and local area will see a yearly economic impact of more than $20.6 million from this companys new payroll.

"North Carolina has been a world leader in biotechnology for many years, but were not resting on our past accomplishments, said North Carolina Commerce Secretary Anthony M. Copeland. Beam Therapeutics joins a host of gene therapy companies that are keeping North Carolina at the forefront of this new frontier of medicine.

Beam Therapeutics project in North Carolina will be facilitated, in part, by a Job Development Investment Grant (JDIG) approved by the states Economic Investment Committee earlier today. Over the course of 12 years, the project is estimated to grow the states economy by $1.36 billion. Using a formula that takes into account the new tax revenues generated by the new jobs, the agreement authorizes the potential reimbursement to the company of up to $3,237,750, spread over 12 years. Payments for all JDIGs only occur following performance verification by the departments of Commerce and Revenue that the company has met its incremental job creation and investment targets. JDIG projects result in positive net tax revenue to the state treasury, even after taking into consideration the grants reimbursement payments to a given company.

Because Beam Therapeutics chose a site in Durham County, classified by the states economic tier system as Tier 3, the companys JDIG agreement also calls for moving as much as $1,079,250 into the states Industrial Development Fund Utility Account. The Utility Account helps rural communities finance necessary infrastructure upgrades to attract future business. Even when new jobs are created in a Tier 3 county such as Durham, the new tax revenue generated through JDIG grants helps more economically challenged communities elsewhere in the state. More information on the states economic tier designations is available here.

In addition to the North Carolina Department of Commerce and the Economic Development Partnership of N.C., other key partners on this project were the the North Carolina Community College System, the North Carolina Biotechnology Center, Durham County, and the Greater Durham Chamber of Commerce.

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Governor Cooper Announces Genetic Medicine Company Will Create 201 Jobs in Durham County - NC Dept of Commerce

CAMBRIDGE, Mass. and GAINESVILLE, Fla., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and the University of Florida today announced a strategic collaboration to enable cutting-edge research for novel genetic medicines. Through the agreement, Sarepta will fund multiple research programs at the University, and will have an exclusive option to further develop any new therapeutic compounds that result from the funded research programs.

We have developed a productive incubator approach to our pipeline development, partnering with the best and brightest in genetic medicine, including leading academic researchers like those at the University of Florida, to discover and translate into meaningful therapies genetic medicine for rare diseases, said Sarepta President and CEO Doug Ingram. Weare excited topartner with andsupportUF research that has the potentialto profoundlyimproveand extend the lives of patients with rare genetic-based diseases.

Through the collaboration, currently unique to UF, funding has been allocated for four innovative projects. These projects include exploratory research in novel gene therapy vectors, next-generation capsids and gene editing technologies as well as work in new therapeutic areas in degenerative genetic diseases. The goal is to foster early relationships with experts and accelerate the scientific advancements that lead to the development of transformational precision genetic medicines for patients in need.

Our researchers intend to find solutions for diseases that have no cure or limited therapeutic options. Their goal is to move these solutions from their labs to patients who need them to see their discoveries change lives. Because Sarepta has a focus and expertise in disease areas that coincide with the work of some of our scientists, its a match and collaboration that make sense and, we hope, will save lives, said Jim OConnell, assistant vice president of UF Innovate, the technology commercialization arm of the university. Sarepta has a bold vision for transforming genetic disease because the company, ultimately, serves patients. That end goal drives its willingness and ability to translate research into a medical reality. We want to be part of that.

University of Florida is a gene therapy powerhouse. UF researchers were the first to discover the life cycle of the adeno-associated virus (AAV), the smallest human virus. Using AAV as a benign delivery vehicle to carry therapeutics to a target, UF was first to reverse blindness in dogs with genetic disease, and UF researchers were integral in the first gene therapy approved by the FDA to treat an inherited genetic disease that can cause blindness. Today, UF is developing technologies in manufacturing, capsid design and therapies to address neuromuscular, cardiovascular, inflammatory, metabolic, pulmonary, skeletal, ophthalmic, and other disorders.

About SareptaAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Sarepta Forward-Looking Statements This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the ability of the collaboration between Sarepta and UF to engage in cutting-edge research for novel genetic medicines; Sareptas commitment to fund multiple research programs at UF; Sareptas option to further develop any new therapeutic compounds that result from the funded research programs; Sareptas incubator approach to discover and translate into meaningful therapies genetic medicine for rare diseases; the collaborations potential to profoundly improve and extend the lives of patients with rare genetic-based diseases; the collaborations ability to foster early relationships with experts to accelerate the scientific advancements that lead to the development of transformational precision genetic medicines; and Sareptas vision to transform genetic disease and translate research into a medical reality.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and UF may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in the discovery of any new therapeutic compounds or any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Contacts:

Sarepta Therapeutics Investors: Ian Estepan, 617-274-4052, iestepan@sarepta.comMedia: Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

UF Innovate: Sara Dagen, 352-294-0998, saradagen@ufl.edu

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Sarepta Therapeutics and University of Florida Announce Collaboration to Accelerate the Discovery and Development of Therapies for Rare Genetic...

Strong Pharmacokinetic and Pharmacodynamic Data Presented in March Validate Platform and Position Company for Scalable Output of Synthetic Precision Genetic Medicines

Company Continues to Progress Candidates in Huntingtons Disease (HD) and Myotonic Dystrophy (DM1)

PITTSBURGH, Aug. 13, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (NeuBase or the Company), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies using its scalable PATrOL platform to address genetic diseases, today reported its financial results for the three and nine month periods ended June 30, 2020.

We are pleased with the continued execution of our development programs during 2020. This includes the announcement in late-March of compelling data that firmly validate our platform as a viable fully synthetic approach to genetic medicine, said Dietrich A. Stephan, Ph.D., chief executive officer of NeuBase. Notably, these data confirm that our therapies penetrate into the brain when administered systemically overcoming one of the grand challenges of drug delivery. PATrOL-enabled compounds can also access tissues throughout the entire body, opening our platform up to unexplored indications that have not previously been accessible by genetic medicine technologies. These positive pharmacokinetic and pharmacodynamic data position our unique technology to output a vast pipeline of therapeutics to resolve innumerable human diseases. We anticipate presenting additional new data with respect to our ongoing progress in the fourth calendar quarter of this year.

A key objective for our company shortly after the March data announcement was to strengthen our balance sheet in order to fully advance our strategies in HD and DM1, and build out our pipeline. This was accomplished in April with the closing of our oversubscribed capital raise of approximately $33.3 million in net proceeds that was led by fundamental healthcare investors and significantly increased our institutional shareholder base. We expect this to support our R&D and general corporate expenses into the second calendar quarter of 2022, continued Dr. Stephan.

Third Fiscal Quarter of 2020 and Recent Operating Highlights

Financial Results for the Fiscal Quarter Ended June 30, 2020:

Financial Results for the Nine Month Period Ended June 30, 2020:

About NeuBase TherapeuticsNeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic diseases.

Use of Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements include, among other things, statements regarding the Companys goals and plans and expectations regarding the timing of our Huntingtons disease (NT0100) and myotonic dystrophy type 1 (NT0200) programs, our capital and liquidity outlook, as well as expanding our pipeline and the potential for the Companys technologies generally. These forward-looking statements are distinguished by use of words such as will, would, anticipate, expect, believe, designed, plan, or intend, the negative of these terms, and similar references to future periods. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Companys current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Companys plans to develop and commercialize its product candidates; the timing of initiation of the Companys planned clinical trials; the timing of the availability of data from the Companys clinical trials; the timing of any planned investigational new drug application or new drug application; the Companys plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Companys product candidates; the Companys commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Companys ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCdaniel@lifesciadvisors.comOP: (617) 430-7576

NeuBase Media Contact:Cait Williamson, Ph.D.LifeSci Communicationscait@lifescicomms.com OP: (646) 751-4366

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NeuBase Therapeutics Reports Financial Results for the Third Fiscal Quarter of 2020 - GlobeNewswire

LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today reported financial results for the quarter ended June 30, 2020, provided a business update and announced the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for LB-001 for the treatment of methylmalonic acidemia in pediatric patients. LogicBio released a separate press release this morning providing further details on the planned Phase 1/2 clinical design for LB-001.

We are thrilled to have received clearance to move forward with this first-in-human clinical trial with our lead product candidate, LB-001, for the treatment of methylmalonic acidemia, a life-threatening congenital genetic disease with no current therapeutic treatment options. This represents a significant milestone in our goal of bringing a treatment to MMA patients as well as for our GeneRide platform. We have maintained continuous dialogue with the centers of excellence that are planned to participate in the Phase 1/2 clinical trial, and we look forward to activating these sites as quickly as possible, said Fred Chereau, CEO of LogicBio. We have instituted systems attempting to mitigate COVID-19 dynamics on our study start-up process and, based on our best estimates, we plan to enroll our first patient in early 2021.

Commenting on the Next Generation Capsid Program, Mr. Chereau said, We are very excited about the recent advances in our novel capsid program, which has generated liver-tropic capsids intended for use in gene editing technologies such as GeneRide and other gene therapy approaches. We are focused on executing across all of our programs and look forward to sharing further details on our novel capsids in early 2021.

Appointment of Daniel Gruskin, M.D. to SVP, Head of Clinical Development

Daniel Gruskin, M.D. was appointed as SVP, head of clinical development in August 2020. Dr. Gruskin has served as interim head of clinical development of LogicBio since June 2020. In April 2020, Dr. Gruskin started consulting with the Company as a special advisor. Previously, Dr. Gruskin served in roles of increasing responsibility at Sanofi Genzyme, most recently as vice president, head of global medical affairs, rare disease, in which capacity he oversaw medical affairs, life cycle management, scientific affairs and other medical and development activities related to metabolic, rare and/or genetic diseases. Prior to his role at Sanofi Genzyme, Dr. Gruskin served as assistant professor, human genetics and pediatrics at Emory University School of Medicine, where he was also the chief of the genetics section at Childrens Healthcare of Atlanta.

Daniel has been instrumental in leading LB-001 clinical development efforts including getting the IND cleared. His deep experience in genetic medicines and metabolic diseases will serve LogicBio well as we look to execute on our goals for both the GeneRide and Next Generation Capsid platforms in search of transformative medicines, said Mr. Chereau.

Anticipated Milestones for 2020 and 2021:

Second Quarter 2020 Financial Results

Three Months Ended June 30, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms.

LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the Companys plans to initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of LB-001 in MMA; the timing, progress and results of the Companys research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; its plans for LB-301 in Crigler-Najjar; and the sufficiency of its cash and cash equivalents to fund operating expenses and capital expenditure requirements. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Investors:Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Media:Stephanie SimonTen Bridge CommunicationsStephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the...

Dr. Richard J. Gralla discusses some of the studies in supportive care that have been put on hold due to the ongoing COVID-19 pandemic.

The onset of the COVID-19 pandemic has forced oncology centers to transition into intensive care units, while oncologists are making emergency rounds. As a result, this has also put a halt on emerging research in supportive care.

In an interview with CURE, Dr. Ricahrd J Gralla details some of the interesting studies he and his colleagues at the Albert Einstein College of Medicine had to put on hold. Studies ranging from toxicities associated with PD-1 and PDL-1 agents in patients with lung cancer to looking at the timing of treatment and when they could switch treatment depending on side effects.

Transcription:

Most institutions, and for ours, studies that we were doing in supportive care in cancer were put on complete hold. And some studies that we were looking at, to really simplify antiemetics in many settings and really home in and prove we've had to put that on hold. There were some interesting areas that we were looking at where we were trying to predict, using some very interesting information, toxicities with anti PD-1 PDL-1 agents, could we predict from a genetic profile who's more likely to have those side effects and also do the same genes that we were looking at, predict the likelihood of response. That's partially based on the immune-genetics of thyroid disease, and we're trying to combine that. So, I hope we can get back to that fairly soon our chief of medicine at Montefiore. Dr. Yale, Tomer is an expert in this area. And as an immunologic endocrinologist, he's been very helpful to our thinking in this way into guiding some of our work.

Also, we've been looking at, can we tell within just two cycles of treatment within six weeks, who's likely to be benefiting from treatment and who is not? And is that a time early on to be able to make a change, and it's looking way beyond imaging, but also using patient reported outcomes to try to predict these things very early on. So far, the results are very encouraging.

So maybe we can get back to all of those when we have less COVID in our midst.

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COVID-19 Puts Supportive Care Studies on Hold for Patients With Lung Cancer - Curetoday.com

News Release

Wednesday, August 12, 2020

According to a new study, mortality rates from the most common lung cancer, non-small cell lung cancer (NSCLC), have fallen sharply in the United States in recent years, due primarily to recent advances in treatment.

The study was led by researchers at the National Cancer Institute (NCI), part of the National Institutes of Health. The findings were published August 12, 2020, in the New England Journal of Medicine.

Reduced tobacco consumption in the U.S. has been associated with a progressive decrease in lung cancer deaths that started around 1990 in men and around 2000 in women.Until now, however, we have not known whether newer treatments might contribute to some of the recent improvement, said Douglas R. Lowy, M.D., NCI deputy director and co-author of this study. This analysis shows for the first time that nationwide mortality rates for the most common category of lung cancer, non-small cell lung cancer, are declining faster than its incidence, an advance that correlates with the U.S. Food and Drug Administration approval of several targeted therapies for this cancer in recent years.

In this study, researchers looked at data for both NSCLC, which accounts for 76% of lung cancer in the U.S., and small-cell lung cancer (SCLC), which accounts for 13% (other subtypes of lung cancer that constitute the remaining share of cases were not covered in this study). In the last decade, new treatments for NSCLC have become available, including those that target genetic changes seen in some NSCLC tumors as well as immune checkpoint inhibitors that help the immune system better attack NSCLC. In contrast, there have been limited treatment advancements for SCLC.

Although death records do not distinguish between lung cancer deaths attributable to NSCLC versus SCLC, the cancer diagnosis records compiled by NCIs Surveillance, Epidemiology, and End Results (SEER) cancer registry program do distinguish between these two subtypes of lung cancer. Therefore, the researchers were able to estimate lung cancer mortality trends for these specific lung cancer subtypes by linking the lung cancer death records for each patient back to the incidence data for these patients in the SEER cancer database.

The researchers found that, in recent years, deaths from NSCLC decreased even faster than the decrease in NSCLC incidence and the decrease in deaths was associated with a substantial improvement in survival. Among men, for example, deaths from NSCLC decreased 3.2% annually from 2006 to 2013 and 6.3% annually from 2013 to 2016, whereas incidence decreased 1.9% annually during 2001 to 2008 and 3.1% annually from 2008 to 2016.

Two-year survival for men with NSCLC improved over this time, from 26% for patients diagnosed in 2001 to 35% for those diagnosed in 2014. Similar improvement was observed for women. In addition, improvements in two-year survival were seen for all races/ethnicities, despite concerns that the newer cancer treatments, many of which are expensive, might increase disparities.

The researchers had originally considered the possibility that lung cancer screening might help explain the decreases in NSCLC mortality, but their findings suggest that lung cancer screening rates, which remained low and stable, do not explain the mortality declines. Instead, the rapid decline in deaths reflects both declines in incidence (due in large part to reductions in smoking) and improvement in treatment.

In contrast, the decrease in deaths from SCLC corresponded with the decrease in incidence, and two-year survival was largely unchanged. Among men, for example, deaths declined 4.3% annually and incidence 3.6% annually. Findings were similar among women. The reduced mortality from SCLC over time, therefore, primarily reflects declines in incidence again, due largely to reduced smoking.

The researchers note that the accelerating decline in NSCLC mortality that began in 2013 corresponds with the time when clinicians began routinely testing patients for genetic alterations targeted by newly approved drugs. In 2012, the National Comprehensive Cancer Network recommended that all patients with nonsquamous NSCLC undergo genetic testing. Subsequently, genetic testing for EGFR (epidermal growth factor receptor) mutations and ALK (anaplastic lymphoma kinase) gene rearrangements which are targeted by the newer treatments increased substantially. Because immune checkpoint inhibitors were not in widespread use over the period of the analysis, the authors suspect that most of the survival benefit was attributable to effective EGFR or ALK inhibitors or other advances in therapy. The effect of immune checkpoint inhibitors on NSCLC survival is significant, which suggests that this improving trend in survival should continue beyond 2016.

The survival benefit for patients with non-small cell lung cancer treated with targeted therapies has been demonstrated in clinical trials, but this study highlights the impact of these treatments at the population level, said Nadia Howlader, Ph.D., of NCIs Division of Cancer Control and Population Sciences, who led the study. We can now see the impact of advances in lung cancer treatment on survival.

About the National Cancer Institute (NCI): NCI leads the National Cancer Program and NIHs efforts to dramatically reduce the prevalence of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about cancer, please visit the NCI website at cancer.gov or call NCIs contact center, the Cancer Information Service, at 1-800-4-CANCER (1-800-422-6237).

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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New treatments spur sharp reduction in lung cancer mortality rate - National Institutes of Health

People who have recovered from COVID-19 can safely interact with others for three months, according to a recent update from the Centers for Disease Control and Prevention suggesting that immunity to the virus may last at least that long.

The recent change is part of the agency's guidance on quarantining. It states that people should quarantine if they've been in close contact with someone who has COVID-19, "excluding people who have had COVID-19 within the past 3 months." People who have tested positive for the virus don't need to be tested again for up to three months, as long as they don't develop symptoms again.

Full coverage of the coronavirus outbreak

The CDC previously acknowledged that people who have recovered from COVID-19 can test positive for the virus for up to three months, though these positive results don't mean that a person is still sick. Instead, the test may be picking up fragments of the virus's genetic code. Dr. Brett Giroir, the undersecretary of health who leads coronavirus testing for the White House, has advised people against being tested again after they recover.

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However, the update goes one step further, suggesting that people who have recovered are protected from getting sick again for at least three months.

The guidance is based on studies that found that, after three months, there was no evidence of people getting re-infected after recovering, a CDC official said. Longer-term studies are needed to determine how long protection might last, and it's still too soon to say to whether a person who has had COVID-19 and recovered is immune.

The update, which was posted on the CDC's website on Aug. 3, is consistent with CDC's earlier guidance not to retest patients within 90 days of an initial infection, Dr. Annie Luetkemeyer, a professor of medicine at the University of California, San Francisco, said. Although some doctors and patients have raised concerns about the possibility of being reinfected with coronavirus, there have been no confirmed reports of reinfection.

Dr. Joshua Barocas, an assistant professor of medicine at the Boston University School of Medicine, said the CDC's update "aligns with the idea that it is unlikely that people can be infected within a three month time frame."

"Of course, unlikely doesn't mean it is impossible to get reinfected," Barocas said, adding that people should be cautious and quarantine "unless it's a crystal clear case."

He noted that the CDC's guidance shouldn't be over-interpreted "an an indication that we have or could soon achieve herd immunity."

"We are not there and we shouldn't rely on that as it will cause a significant amount of mortality."

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Sara G. Miller is the health editor for NBC News, Health & Medical Unit.

Akshay Syalis a medical fellow with the NBC News Health and Medical Unit.

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CDC suggests recovered COVID-19 patients have protection for 3 months - NBC News

Researchers at Weill Cornell Medicine Qatar (WCM-Q) have helped to shine a light on traits that are associated with various degrees of consanguineous marriage.With funding from the Biomedical Research Program at WCM-Q, Dr. Steven C. Hunt, professor of genetic medicine, and Dr. Noha A. Yousri, adjunct assistant professor of research in genetic medicine, participated in a large meta-analysis with hundreds of scientists from across the globe, studying genetic variations in more than 1.4 million individuals.Dr. Yousri said: Very large numbers of samples are required to study the impact of marriage between men and women from the same extended families. This study conducted a meta-analysis of results from 119 independent cohorts to quantify the effect of consanguineous marriages on 45 commonly measured complex traits of biomedical or evolutionary importance, and an additional 55 more rarely measured traits included in the UK Biobank. Complex traits were arranged into 16 groups covering major organ systems and disease risk factors were analyzed.

Genetic associations with 32 of 100 studied traits and conditions in humans were identified by using runs of homozygosity (ROH). ROH refers to long continuous segments of identical alleles (homozygosity) along both inherited chromosomes. ROH arise through consanguineous marriages, i.e., when both parents are related through a common ancestor. The net directional effect of all recessive variants on traits is quantified and related to the 100 traits and conditions

Dr. Hunt said: Runs of rare recessive variants inherited through these types of unions were shown to reduce reproductive fitness or fertility, with a 55% reduction in the odds of having children. ROH were also associated with decreased height, increased waist to hip ratio, decreased lung function, and reduced educational attainment.

Dr. Yousri said: The most important aspect of this study is finding the traits affected by consanguineous marriages. Given that this type of marriage is relatively common in Qatar, it would be beneficial to investigate whether those results might apply to Qatar, as it might help us better understand the implications.The full study, which was led by Professor James F. Wilson and his group from the University of Edinburgh, has been published in the high impact journal Nature Communications, and can be read at https://www.nature.com/articles/s41467-019-12283-6.

Dr. Yousri is also collaborating on a related study with Sidra Medicine called The PMED-Qatar Study: Personalized Molecular Evaluation and Diagnosis for Rare Diseases in Qatar, which is being funded by Qatar National Research Fund under the project number NPRP11S-0110-180250.

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Scientists at WCM-Q Examine the Implications of Consanguineous Marriage - Al-Bawaba

A startup developing precision health technology for cancer has raised its first major round of venture capital funding.

Chicago-based CancerIQ said Thursday that it had raised a Series A funding round of $4.8 billion, led by HealthX Ventures, which is a digital health-focused venture capital firm. The company said it plans to use the money to further growth of its product offering and integration with electronic health records and genetic testing partners.

CancerIQs technology is focused on allowing hospitals to use genomics to personalize the prevention and early detection of cancers. Designed to integrate easily into the clinical workflow, the platform helps providers identify, evaluate and manage populations based on genetic risk factors while also enabling virtual visits.

Our mission is really to predict and preempt hereditary diseases, starting off with those that are most prevalent in our community, said CancerIQ CEO Feyi Ayodele in a phone interview. Diseases that are of particular interest include hereditary breast, ovarian and colon cancer, as well as familial hypercholesterolemia, she added.

The company said its workflows allow health systems to use precision health strategies for patients predisposed to cancer by identifying the 25% of those who qualify for genetic testing; streamlining the genetic testing and counseling process, over telehealth if required; managing high-risk patients over time; and tracking outcomes at the individual and population levels.

Partnering is of interest as well, in diagnostics as well as life sciences.

If you think about it, there are a number of innovations out there billions raised for genetic testing companies and liquid biopsy companies and companies that are producing targeted therapies, Ayodele said. The challenges they all face are provider knowledge that patients are appropriate for that therapy and ease of use for providers to actually take advantage of those innovations that are out there.

The news closely follows the release on Aug. 6 of a report by the University of Pittsburgh Medical Centers Center for Connected Medicine, showing that most hospital and health systems foresee an increase in genomics and genetics vendors in 2023, with nine-in-10 saying they were already providing genomic or genetic testing or were planning to do so.

Photo: claudenakagawa, Getty Images

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CancerIQ raises $4.8M Series A funding round for cancer precision health technology - MedCity News