Category Archives: Genetic Medicine

LONDON 10 June, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO (ivacaftor) to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older and weighing at least 5 kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene the most common residual function mutation underlying CF.

A little over eight years ago, KALYDECO was approved as the first and only medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations, said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. Since then, its been our goal to ensure that as many people with CF as possible are able to benefit from our treatments, and todays label extension means that approximately 500 young patients in Europe, who have long awaited a treatment option, are now eligible for KALYDECO.

Now approved, KALYDECO (ivacaftor) will be immediately available to additional eligible patients in Germany and shortly in countries where respective long-term reimbursement agreements have been previously secured. Vertex will work closely with all other relevant government authorities to secure access for eligible patients as quickly as possible.

In Europe, KALYDECO (ivacaftor) is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and in infants ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

About KALYDECO (ivacaftor)

Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in theCFTRgene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

About Vertex

Vertex 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 inCambridge, Mass., Vertex's global headquarters is now located inBoston'sInnovation Districtand its international headquarters is inLondon, UK. Additionally, the company has research and development sites and commercial offices inNorth America,Europe,AustraliaandLatin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years onScience magazine'sTop Employers list and top five on the 2019 Best Employers for Diversity list byForbes.

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Vertex Announces European Commission Approval for KALYDECO (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months...

AMES, Iowa A new study that looks closely at the cardiac health of flies provides new evidence that liver dysfunction may lead to deterioration of the heart.

The research fills in gaps in how scientists understand the links between heart health and other tissues and could inform the development of new therapies in human medicine, said Hua Bai, an assistant professor of genetics, development and cell biology at Iowa State University. Bais lab has performed previous studies on how cardiac health in flies changes with age. The new study, published in the academic journal Nature Communications, also covers new ground in the function of a poorly understood organelle called the peroxisome, which may play a major role in how organisms age.

We were thinking outside the heart for this paper, Bai said. We wanted to find out if other tissues affect cardiac function during aging. There is significant data suggesting that liver function actually is a risk factor for cardiac disease. A patient with a lot of liver dysfunction often develops cardiac disease. This is a concern because you may have two diseases that you have to deal with for these patients.

But Bai said no direct link between liver and heart disease has emerged in experiments, leaving medical professionals unsure if the two factors share a causal relationship or if theres simply a correlation. Bais lab attempted to fill that gap by studying the interaction between liver disease and the function of cardiac muscles in flies.

Previous studies from Bais lab showed that manipulating genes in the cardiac muscles of flies could restore the heart function of older flies to a state similar to younger flies, essentially turning back the clock on cardiac tissues. In the new experiments, the researchers manipulated various genes governing liver function in flies to see how that would affect heart health as the flies aged.

Our findings demonstrate we can protect the liver of old animals and maintain the health of the heart without doing any direct intervention on the heart tissue, said Kerui Huang, a graduate student in Bais laboratory and the lead author of the study.

Much of the genetic work the researchers conducted focused on peroxisomes, understudied organelles inside cells that regulate key lipid metabolic processes and detoxification critical for brain and liver function.

Looking at all the biology literature, we dont know much about how peroxisome function changes in aged animals, Bai said. We show that peroxisomal protein import function is significantly impaired in aged flies. Research like ours could open up another new field to study how peroxisomes regulate tissue aging.

Huang said although flies appear to be highly dissimilar to humans, human medicine still has much to gain from studying fly biology. For instance, the functions of a flys liver and heart share many similar functions with the human liver and heart.

Pharmaceutical companies have shown great interest in finding new avenues to treat age-related disease, Bai said. The relationship between peroxisomes, liver function and heart aging described in the new study might become a promising target for new therapies and drugs, he said.

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Link between liver and heart disease could lead to new therapeutics News Service Iowa State University - Iowa State University News Service

As a physician, I realized that the current method of trial and error for prescribing medications can be substantially improved using genomics

SEATTLE (PRWEB) June 11, 2020

ActXs genetic clinical decision support service, also known as GenoACTTM, helps physicians provide better care using the patients genetic data. Today, ActX announced that the U.S. Patent and Trademark Office has issued Patent No. 10,586,612. It covers methods and systems for organizing, storing, searching, aggregating, and distributing large amounts of biological information obtained for individual patients. With ActX, once patients are genotyped or sequenced, their data is stored in the cloud and referenced in real time. This provides physicians with real time decision support as they use their Electronic Medical Record. For example, if a physician writes a prescription for a medication that may not be effective, then based on the patients genetics, ActX will provide an alert in their normal workflow. The alert has minimal latency, succinctly informs the physician, and provides suggestions for more effective treatment.

The patent covers the use of electronic biological information, stored and distributed through a cloud-like information storage facility to patients, clinicians, and electronic health record-based applications. The ability to provide patients and their physicians with actionable information quickly in real time, based on a large amount of genetic data, in normal physician workflow, is new to the healthcare industry. This was developed by ActXs CEO, Dr. Andrew Ury, and Software Engineer Michael Arlt. The 612 Patent covers 21 claims and is valid through March 3rd, 2034.

The service we have created with ActX helps make genomics actionable, a key part of which is pharmacogenomics, in everyday medical practice, said Dr. Andrew Ury, founder of ActX. As a physician, I realized that the current method of trial and error for prescribing medications can be substantially improved using genomics. Instead of trying out a medication for months to see if it works or causes serious side effects, physicians and patients can, for many medications, using genetics, know in advance whether a medication is likely to work or cause side effects. The approval of this patent allows us to continue with our mission of making real-time genomic decision support a standard in patient care.

This newly allowed patent is owned by ActX, with the inventors being Andrew Ury and Michael Arlt. It is one of the latest U.S. patents issued in connection with precision medicine and cloud-based information storage and sharing.

About ActX

ActX is the industry leader in EHR-integrated precision medicine. Our service helps physicians make better decisions about medical treatment, using a patients genetic information to guide therapy. The ActX Genomic Service offers proven, tightly integrated, real-time genomic decision support for medication orders and actionable genomic risks, a built in patient Genomic Profile, and the ability to fully customize content.

As medications are ordered, and before the prescription is finalized, medications are checked against the patients genetics for efficacy, adverse reactions, and dosing. Extensive evidence based and actionable content is provided as default, customizable content, covering hundreds of genes. You can easily suppress any content or interactions, or add or modify content. The ActX Genomic Service is live at numerous U.S. health systems. For more information, visit http://www.actx.com, email info@actx.com or call 888-998-2289.

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ActX Announces Issuance of US Patent for Real-Time Genomic Decision Support - PR Web

NEW YORK, June 11, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (Fortress), an innovative biopharmaceutical company focused on acquiring, developing and commercializing high-potential marketed pharmaceutical products and development-stage pharmaceutical product candidates, today announced the publication of a study, Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database (gnomAD), in Molecular Genetics and Metabolism Reports. The study was published online in June 2020 and will be published in the print edition of the journal in September 2020.

The study evaluated the prevalence of Menkes disease, an often lethal, if untreated, X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase. Previous estimates of Menkes disease were based on confirmed clinical cases ascertained from specific populations and varied from 1 in 40,000 to 1 in 354,507.

Led by Stephen G. Kaler, M.D., M.P.H., a physician-scientist in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children's Hospital, the authors reviewed the canonical ATP7A transcript in the current version of gnomAD (v2.1.1) to evaluate frequencies of loss-of-function and pathogenic missense variants in a diverse normal control population. Assuming Hardy-Weinberg genetic equilibrium, the allelic frequency of loss-of-function variants suggests a minimum birth prevalence for Menkes disease of 1 in 34,810 males, higher than previously recognized. If likely pathogenic missense variants are included, the estimated birth prevalence could potentially be as high as 1 in 8,664 live male births.

Based on these findings, it appears that Menkes disease is under-reported in the population. This may reflect disparities in access to health care or tertiary care genetics clinics, challenges in distinguishing the Menkes phenotype from other conditions, and deaths of affected subjects before diagnosis, said Dr. Kaler, also a professor of Pediatrics and Genetics at The Ohio State University College of Medicine.

Our latest study suggests that a newborn screening pilot study for Menkes disease would confirm a higher than previously estimated prevalence, as we have seen with other rare inherited disorders such as Pompe disease, said Lung S. Yam, M.D., Ph.D., President and Chief Executive Officer of Cyprium. Earlier diagnosis of Menkes disease through newborn screening would likely increase the number of Menkes disease patients identified at birth and allow for institution of early treatment during the asymptomatic phase of the condition.

The study can be accessed here.

About Menkes Disease and Related Copper Metabolism DisordersMenkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of copper transporter ATP7A. Biochemically, Menkes patients have low levels of copper in their blood and brain, as well as abnormal levels of certain neurochemicals. Definitive diagnosis is typically made by sequencing the ATP7A gene. The condition is characterized by distinctive clinical features, including sparse and depigmented hair (kinky hair), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, and failure to thrive. Mortality is high in untreated Menkes disease, with many patients dying before the age of three. Milder versions of ATP7A mutations are associated with other conditions, including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants.

About CUTX-101 (Copper Histidinate)CUTX-101 is in clinical development to treat patients with Menkes disease by replenishing Copper Histidinate, restoring copper homeostasis, and maintaining serum copper levels in the normal age appropriate range. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under cGMP that is intended to improve tolerability due to physiological pH and to bypass the oral absorption of copper, which is impaired in patients with Menkes disease. In a Phase 1/2 clinical trial conducted by Stephen G. Kaler, M.D., M.P.H., at the National Institutes of Health (NIH), early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. A Phase 3 trial of CUTX-101 in patients with Menkes disease also led by Dr. Kaler has completed enrollment. A Cyprium-sponsored expanded access protocol for Menkes disease patients is ongoing.

About Cyprium TherapeuticsCyprium Therapeutics, Inc. (Cyprium), is focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders. In March 2017, Cyprium entered into a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), part of the National Institutes of Health (NIH), to advance the clinical development of CUTX-101 (Copper Histidinate injection) for the treatment of Menkes disease. In addition, Cyprium and NICHD entered into a worldwide, exclusive license agreement to develop and commercialize adeno-associated virus (AAV)-based gene therapy, called AAV-ATP7A, to deliver working copies of the copper transporter that is defective in Menkes patients, and to be used in combination with CUTX-101. CUTX-101 was granted U.S. Food and Drug Administration (FDA) Fast Track and Rare Pediatric Disease Designations, and both CUTX-101 and AAV-ATP7A have received FDA Orphan Drug Designation previously. Cyprium was founded by Fortress Biotech, Inc. (Nasdaq: FBIO) and is based in New York City. For more information, visit http://www.cypriumtx.com.

About Fortress Biotech Fortress Biotech, Inc. (Fortress) is an innovative biopharmaceutical company that was recently ranked number 10 in Deloittes 2019 Technology Fast 500, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors, based on percentage of fiscal year revenue growth over a three-year period. Fortress is focused on acquiring, developing and commercializing high-potential marketed pharmaceutical products and development-stage pharmaceutical product candidates. The company has five marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and at partners it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is driven by a world-class business development team that is focused on leveraging its significant biopharmaceutical industry expertise to further expand the companys portfolio of product opportunities. Fortress has established partnerships with some of the worlds leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including Alexion Pharmaceuticals, Inc., AstraZeneca, City of Hope, Fred Hutchinson Cancer Research Center, InvaGen Pharmaceuticals Inc. (a subsidiary of Cipla Limited), St. Jude Childrens Research Hospital and Nationwide Childrens Hospital. For more information, visitwww.fortressbiotech.com.

Forward-Looking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. As used below and throughout this press release, the words we, us and our may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein.

Company Contacts:Jaclyn Jaffe and William BegienFortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com

Lung Yam, M.D., Ph.D.Cyprium Therapeutics, Inc. ir@cypriumtx.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Fortress Biotech Announces Publication of Study on Estimated Birth Prevalence of Menkes Disease in Molecular Genetics and Metabolism Reports -...

XconomyBoston

Verve Therapeutics aims to use gene editing to address heart attacks by targeting the liver, not the heart. And it wants to make its edits inside the patient. The startups approach raises a lot of questions. Verve has some answers now, and it has raised $63 million to learn more.

The new financing announced Thursday was led by GV, the same investor that led the Cambridge, MA-based companys Series A round of funding last year. With the additional cash, Verve CEO Sekar Kathiresan says Verve could reach its first human tests of a gene-editing therapy in about three years.

High cholesterol and triglyceride levels in the blood are among the biggest risk factors for a heart attack. For some people, high levels of these types of fats are inherited. In a condition called familial hypercholesterolemia, a genetic defect makes it harder for these people to remove the bad form of cholesterol from their blood. No matter how much they do in terms of diet and exercise, they continue to have high cholesterol levels that raise the risk of a heart attack.

Low cholesterol levels can also be inherited. In rare cases, people have a genetic mutation that turns off the gene that leads to high levels of cholesterol, Kathiresan says. Verves research is built on the 15 years of genetic research that identified eight genes linked to heart health. Such research has led to the commercialization of drugs that block PCSK9, a protein that inhibits the bodys ability to clear the bad form of cholesterol. But these drugs must be taken chronically.

Gene editing is typically thought of as a way to correct a genetic mutation that causes a problem. Verve, instead, is trying to give patients a mutation that confers a benefitturning off the gene that leads to high levels of cholesterol and triglycerides. If it works, its a one-time treatment.

What youre left with is the gene turned off forever, Kathiresan says. Its durable, lifelong control of cholesterol.

The genetic alteration would be made in vivoinside the patientby messenger RNA (mRNA), which is a genetic blueprint that carries editing instructions, and a guide RNA that gets this editor to the target gene. But to make the intended edit, the two pieces of nucleic acid need to reach their destination intact. Some companies are employing viral delivery systems. Thats the approach taken by Editas Medicine (NASDAQ: EDIT) and Allergan, now a subsidiary of AbbVie (NYSE: ABBV). Their experimental in vivo gene-editing therapy for a form of Leber congenital amaurosis, an inherited form of blindness, is delivered to target cells in the eye via an engineered adeno-associated virus. In March, a Phase 1/2 study testing the experimental therapy, AGN-151587, dosed its first patient.

A gene-editing therapy delivered via an engineered virus stays in the patient for months, which increases the risk of edits beyond the intended target, Kathiresan says. Instead, Verve uses a lipid nanoparticle, which stays in the system for about 48 hours. Such particles have been shown to quickly migrate to the liver, where they are easily taken up by the cells of the organ, Kathiresan says. Once in those cells, the Verve therapy does its work to turn off the target gene.

Verve is conducting its research with two gene-editing tools, CRISPR and base editing. Kathiresan says in the past year the company has evaluated combinations of tools and targets to determine which offer the best shot at reducing cholesterol levels while posing the lowest risk. So far the company has conducted tests in mice and monkeys. Kathiresan expects Verve will select a lead candidate by the end of this year. After that, he anticipates 18 months of preclinical research to support an application seeking FDA permission to begin tests of the gene-editing therapy in humans. Kathiresan says the first clinical trial testing Verves gene-editing drug will enroll familial hypercholesterolemia patients.

To date, Verve says it has raised $123 million total. The company calls the latest financing a Series A2 round. Earlier investors ARCH Venture Partners, F-Prime Capital, and Biomatics Capital also participated. Besides adding to its initial round, the Series A2 boosted Verves valuation, Kathiresan says. The new round also adds Wellington Management and Casdin Capital, two so-called crossover investorsfirms that invest in both private and public companies.

Crossover investment is viewed as a sign a company is preparing for an IPO. Kathiresan acknowledges that his company will need to raise more cash down the road but says its too early to determine what form that financing will take. He is, however, preparing to talk more about what Verve has accomplished so far. Kathiresan is the scheduled keynote speaker on June 27 during the annual meeting of the International Society for Stem Cell Research, where he says hell discuss more details about Verves scientific findings.

Image: iStock/SergeyNivens

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan@xconomy.com.

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Verve Therapeutics Adds $63M to Edit Heart Attack Risk Out of Genes - Xconomy

WALTHAM, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies, announced today the appointment of Elliott Sigal, M.D., Ph.D., to the companys Board of Directors. Dr. Sigal has more than 25 years of leadership experience in the biopharmaceutical industry and is the former Chief Scientific Officer and President of R&D for Bristol Myers Squibb.

As a trailblazer in the biopharmaceutical industry, Dr. Sigal has demonstrated a track record of bringing transformative medicines to patients, said Rick Modi, Chief Executive Officer at Affinia Therapeutics. We look forward to the counsel he will provide to advance our platform and investigational product candidates toward the clinic and the patients who need them most.

Dr. Sigal is a former member of the Board of Directors of Spark Therapeutics. During his tenure from 2014 to 2019, the companys lead product, LUXTURNA was approved as the first AAV gene therapy in the United States. The company was acquired by Roche in 2019.

Prior to Spark Therapeutics, Dr. Sigal was an Executive Vice President and Director of Bristol Myers Squibb. While at BMS, he led the team that established BMS at the forefront of immuno-oncology which is revolutionizing the treatment of cancer and brought fourteen new medicines to market for patients with devastating diseases in areas including oncology, hematology, cardiovascular disease, hepatitis, rheumatoid arthritis and neuropsychiatry. Dr. Sigal was instrumental in increasing R&D productivity and developing the companys strategy in biologics. In 2012, he was named the best R&D chief in the pharmaceutical industry by Scrip Intelligence.

Affinia Therapeutics is setting a new standard in gene therapy, said Dr. Elliott Sigal. I am pleased to join the companys board at such an exciting time as they pioneer and design vectors and genetic medicines to transform the applicability of gene therapies for patients in need.

Dr. Sigal received his medical degree from the University of Chicago in 1981 and trained in Internal Medicine and Pulmonary Medicine at the University of California, San Francisco (UCSF). He also holds a Bachelor of Science, Master of Science and Ph.D. in Industrial Engineering from Purdue University. Dr. Sigal currently serves as a senior advisor to the healthcare team of New Enterprise Associates and consults for select biotechnology companies including Amgen. He is co-chair of the Scientific Advisory Board of Amgen and is a member of the Scientific Steering Committee of the Sean Parker Institute for Cancer Immunotherapy. He is also a member of the Board of Directors for the biotechnology companies Adaptimmune and Surface Oncology. Dr. Sigal joined BMS in 1997 and held roles in both discovery and development before ascending to Chief Scientific Officer and President of R&D. Positions prior to BMS included a faculty appointment at UCSF, senior executive roles at Syntex/Roche and CEO of the genomics firm, Mercator Genetics.

Dr. Sigal joins Affinia Therapeutics board which includes Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, New Enterprise Associates; Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School; Rick Modi, Chief Executive Officer, Affinia Therapeutics; Robert Weisskoff, Ph.D., Partner, F-Prime Capital; and Sean Nolan, Chairman of the Board of Directors at Affinia Therapeutics.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need. http://www.affiniatx.com.

Affinia Therapeutics Contacts

Investors: investors@affiniatx.com

Media: media@affiniatx.com

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Affinia Therapeutics Announces Appointment of Elliott Sigal, MD, Ph.D. to the Company's Board of Directors - BioSpace