Newswise PHILADELPHIAInherited mutations in a gene that keeps nerve cells intact was shown, for the first time, to be a driver of a neuropathy known as Charcot-Marie-Tooth (CMT) disease. This finding is detailed in a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania, which published in Neurology Genetics, an official journal of the American Academy of Neurology.

The findings, thanks to siblings treated at Penn since the late 1980s, present a clearer picture of the diseases genetic underpinnings that could inform the development of gene therapies to correct it.

The mutations in the gene known as dystonin (DST) add to a growing list of malfunctions found to cause their type of CMT, known as CMT2, which is defined by the loss of the nerve fibers, or axons, in the peripheral nerve cells. The researchers also showed that these mutations affect two key protein isoforms, BPAG1-a2 and BPAG1-b2, that are involved in nerve fiber function. Mutations in other isoforms of the same protein were previously tied to a blistering skin disease.

Neuropathies are common, occurring in nearly half of all diabetic patients, while hereditary neuropathies affect nearly one of out of 2,000 people. CMT is a debilitating neurodegenerative disorder that usually strikes in the second or third decade of life, and leaves patients with numbness and weakness in the hands and feet, among other neurological-related conditions.

There are more than 100 mutations found to be associated with CMT, with likely many more out there. Past studies from Penn researchers have identified some of these mutations by studying patients treated at Penn Medicine.

We are determined to fill in the blanks of this giant jigsaw puzzle, said senior author Steven S. Scherer, MD, PhD, a professor of Neurology. This latest paper is but one of many examples of where breakthroughs have happened between patients and the doctors at Penn and the support of different organizations and institutions to bring it all together."

The researchers applied whole exome sequencing to analyze the more than 30 million base pairs of DNA that encode the 20,000 proteins in humans. By examining three siblingstwo affected and one unaffectedthe researchers were able to deduce the genetic basis of mutations that caused the two siblings to be affected.

Backed by a mouse model from past studies showing a role of dystonin in neuropathies, the researchers identified two recessive mutations on the DST gene, each received from a biological parent, as the culprit. Together, the two mutations in the affected siblings disrupt the BPAG1-a2 and BPAG1-b2 isoforms, the researchers found, which weakened their axonal health. The DST gene gives rise to proteins that regulate the organization and stability of the microtubule network of sensory neurons to allow for transport of different cellular material along the nerve fibers.

We have collaborated with this family for 30 years, and now we finally have an answer, Scherer said, and the answer was a new genetic cause of neuropathy.

The findings put the field steps closer to developing new targeted therapeutics as well as CMT gene therapies designed to replace missing genes or correct mutations driving the disease. Clinical trials to investigate these latest mutations and others are not far off in the future, the researchers believe, particularly at an institution like Penn, which is home to the second largest clinic for CMT patients in the country and well-known for its gene therapy program.

We are in the era where treatments for genetic diseases are possible, Scherer said. This brother and sister stand to benefit from that approach because we know the gene that is missing, and if we could replace it, that should at least prevent their progression.

Co-authors of the study include William Motley, MD, DPhil, a former medical student at Penn and now of Third Rock Ventures, a venture capital firm that invests in biotechnology, and Stephan Zuchner, MD, PhD, of the department of Human Genetics at the University of Miami.

This work was supported by the Judy Seltzer Levenson Memorial Fund for CMT Research, the INC (U54NS065712), which is a part of the NCATS Rare Diseases Clinical Research Network, an initiative of the Office of Rare Diseases Research, and in part by the National Institutes of Health (R25NS065729).

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

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

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

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

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

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Penn Researchers Identify New Genetic Cause of a Form of Inherited Neuropathy - Newswise

A new meta-analysis tries to shed light on why strokes hit the black population harder than other groups.

Published in Stroke, the COMPASS (Consortium of Minority Population Genome-Wide Association Studies of Stroke) included more than 22,000 individuals of African ancestry from 13 different cohorts (3,734 cases and 18,317 controls).

Stroke is a complex disease with multiple genetic and environmental risk factors, the authors wrote. Blacks endure a nearly 2-fold greater risk of stroke and are 2 to 3 times more likely to die from stroke than European Americans.

The authors identified a single nucleotide polymorphism (rs55931441) near HNF1A gene that they reported had genome-wide significance (P=4.62108) along with at least 29 other polymorphisms at 24 unique loci that they said showed evidence of an association (P<1106). The authors compared this to other populations by performing a look-up analysis in Europeans and Hispanics (in the Stroke Genetics Network). Sixteen of 24 loci across multiple populations validated the findings for individuals of African descent. They also wrote that variants in the SFXN4andTMEM108 genes represented potential novel ischemic stroke loci.

Despite its limitations, the authors noted that genetic studies like COMPASS that include minority populations present potential for identifying underlying stroke disparities. The upshot, they said, was potential advances in precision medicine that would lead to better stroke risk stratification in diverse populations.

Given the undue burden that people of African ancestry endure from stroke and other cerebrovascular disease, the lack of investigation of risk factors in this group has been a substantial gap, said researcher Bradford B. Worrall, MD, a neurologist at UVA Health, in a press release. Our work is an important step toward filling that gap, albeit with much more work to be done. These findings will provide greater insight into ethnic-specific and global risk factors to reduce the second leading cause of death worldwide.

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Genetic Variant in Persons of African Descent Linked with Stroke: Analysis - DocWire News

SAN FRANCISCO, Aug. 4, 2020 /PRNewswire/ --Invitae Corporation (NYSE: NVTA), a leading medical genetics company, today announced financial and operating results for the second quarter ended June 30, 2020.

"While we experienced significant disruptions in the healthcare system due to the pandemic, we quickly established a solid recovery during the quarter. Our results highlight the strength of our operations and the benefits of our diversified menu, investments in telehealth capabilities and longstanding customer relationships, all of which position us to adapt and meet the changing needs of our customers," said Sean George, Ph.D., co-founder and chief executive officer of Invitae. "We exited the quarter with a strong footing and increasing momentum. We remain confident in our ability to continue to deliver on our mission to bring genetic information into mainstream medicine."

Second Quarter 2020 Financial Results

Total operating expense, excluding cost of revenue, for the second quarter of 2020 was $145.3 million. Non-GAAP operating expense was $105.7 million in the second quarter of 2020.

Net loss for the second quarter of 2020 was $166.4 million, or $1.29 net loss per share, compared to a net loss of $48.7 million in the second quarter of 2019, or $0.54 net loss per share. Non-GAAP net loss was $99.2 million, or $0.77 non-GAAP net loss per share, in the second quarter of 2020.

At June 30, 2020, cash, cash equivalents, restricted cash and marketable securities totaled $428.5 million. Net increase in cash, cash equivalents and restricted cash for the quarter was $78.0 million. Cash burn, including various acquisition-related expenses, was $89.2 million for the quarter and $63.8 million when excluding the $25.4 million cash paid to acquire YouScript and Genelex.

Early in the quarter, in response to impacts of the pandemic, the company took actions to significantly scale back operational expenditures. The result of these changes is expected to decrease the discretionary spend in cost of revenue and operating expense beginning in the third quarter.

"We continue to see a solid recovery in volume, improvement in our operating leverage and ability to improve revenue generation. As a result, we are well positioned with sufficient capital to execute our strategy in the coming years," continued Dr. George. "We have added or will be adding important capabilities to our platform through the acquisitions we announced this quarter and our ongoing product development efforts. With our mission clearly in focus, we can continue to navigate these unprecedented times."

Corporate and Scientific Highlights

Webcast and Conference Call DetailsManagement will host a conference call and webcast today at 4:30 p.m. Eastern / 1:30 p.m. Pacific to discuss financial results and recent developments. To register for the conference call and webcast, please use one of the methods below. Upon registering, each participant will be provided with call details and a registrant ID.

Online registration: http://www.directeventreg.com/registration/event/5882896

Phone registration: (888) 869-1189 or (706) 643-5902

The live webcast of the call and slide deck may be accessedhere or by visiting the investors section of the company's website atir.invitae.com. A replay of the webcast and conference call will be available shortly after the conclusion of the call and will be archived on the company's website.

About InvitaeInvitae Corporation(NYSE: NVTA)is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.

Safe Harbor StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the company's belief that it is well-positioned to adapt and meet the changing needs of its customers; the company's belief regarding the momentum of its business and ability to continue to deliver on its mission to bring genetic information into mainstream medicine; the impact of the COVID-19 pandemic on the company's business, and the measures it has taken or may take in the future with respect thereto; the impact of the company's acquisitions, including its proposed merger with ArcherDX, as well as its partnerships and product offerings; and the company's beliefs regarding the growth of its business, its position and impact on the genetic testing industry, its success in executing on its mission and strategy, and the benefits of genetic testing. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the impact of the COVID-19 pandemic on the company, and the effectiveness of the efforts it has taken or may take in the future in response thereto; the company's ability to continue to grow its business, including internationally; the company's history of losses; the company's ability to compete; the company's failure to manage growth effectively; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the risk that the company may not obtain or maintain sufficient levels of reimbursement for its tests; the ability of Invitae and ArcherDX to obtain the approval of Invitae's and ArcherDX's stockholders, and to satisfy the other conditions to the closing of the acquisition and related financing transactions on a timely basis or at all; the occurrence of events that may give rise to a right of one or both of Invitae and ArcherDX to terminate the merger agreement; the company's failure to successfully integrate or fully realize the anticipated benefits of acquired businesses; the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Non-GAAP Financial MeasuresTo supplement Invitae's consolidated financial statements prepared in accordance with generally accepted accounting principles in the United States (GAAP), the company is providing several non-GAAP measures, including non-GAAP gross profit, non-GAAP cost of revenue, non-GAAP operating expense, including non-GAAP research and development, non-GAAP selling and marketing, non-GAAP general and administrative and non-GAAP other income (expense), net, as well as non-GAAP net loss and non-GAAP net loss per share and non-GAAP cash burn. These non-GAAP financial measures are not based on any standardized methodology prescribed by GAAP and are not necessarily comparable to similarly-titled measures presented by other companies. Management believes these non-GAAP financial measures are useful to investors in evaluating the company's ongoing operating results and trends.

Management is excluding from some or all of its non-GAAP operating results (1) amortization of acquired intangible assets, (2) acquisition-related stock-based compensation related to inducement grants, (3) post-combination expense related to the acceleration of equity grants or bonus payments in connection with the company's business combinations, (4) adjustments to the fair value of our acquisition-related liabilities and (5) acquisition-related income tax benefits. These non-GAAP financial measures are limited in value because they exclude certain items that may have a material impact on the reported financial results. Management accounts for this limitation by analyzing results on a GAAP basis as well as a non-GAAP basis and also by providing GAAP measures in the company's public disclosures.

Cash burn excludes (1) changes in marketable securities, (2) cash received from equity financings and (3) cash received from exercises of warrants. Management believes cash burn is a liquidity measure that provides useful information to management and investors about the amount of cash consumed by the operations of the business. A limitation of using this non-GAAP measure is that cash burn does not represent the total change in cash, cash equivalents, and restricted cash for the period because it excludes cash provided by or used for other operating, investing or financing activities. Management accounts for this limitation by providing information about the company's operating, investing and financing activities in the statements of cash flows in the consolidated financial statements in the company's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and by presenting net cash provided by (used in) operating, investing and financing activities as well as the net increase or decrease in cash, cash equivalents and restricted cash in its reconciliation of cash burn.

In addition, other companies, including companies in the same industry, may not use the same non-GAAP measures or may calculate these metrics in a different manner than management or may use other financial measures to evaluate their performance, all of which could reduce the usefulness of these non-GAAP measures as comparative measures. Because of these limitations, the company's non-GAAP financial measures should not be considered in isolation from, or as a substitute for, financial information prepared in accordance with GAAP. Investors are encouraged to review the non-GAAP reconciliations provided in the tables below.

INVITAE CORPORATION

Consolidated Balance Sheets

(in thousands)

(unaudited)

June 30,2020

December 31,2019

Assets

Current assets:

Cash and cash equivalents

$

168,203

$

151,389

Marketable securities

253,933

240,436

Accounts receivable

27,905

32,541

Prepaid expenses and other current assets

21,081

18,032

Total current assets

471,122

442,398

Property and equipment, net

43,381

37,747

Operating lease assets

38,239

36,640

Restricted cash

6,343

6,183

Intangible assets, net

192,644

125,175

Goodwill

211,225

126,777

Other assets

6,921

6,681

Total assets

$

969,875

$

781,601

Liabilities and stockholders' equity

Current liabilities:

Accounts payable

$

20,091

$

10,321

Accrued liabilities

99,490

64,814

Operating lease obligations

6,339

4,870

Finance lease obligations

977

1,855

Total current liabilities

126,897

81,860

Operating lease obligations, net of current portion

42,134

42,191

Finance lease obligations, net of current portion

879

1,155

Convertible senior notes, net

276,092

268,755

Deferred tax liability

10,250

Other long-term liabilities

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Invitae Reports $46.2 Million in Revenue Driven by More Than 120000 Samples Accessioned in the Second Quarter of 2020 - Monterey County Weekly

ByCarly Kempler

Noel R. Rose, a longtime Johns Hopkins Bloomberg School of Public Health faculty member and researcher widely recognized as a father of autoimmune disease research, died Thursday at home in Brookline, Massachusetts. He was 92.

"Noel was an extraordinary scientist and an exceptionally kind and humble human beingalways looking out for the well-being of faculty and students," said Ellen J. MacKenzie, the current dean of the Johns Hopkins Bloomberg School of Public Health. "I admired his steadfast commitment to excellence coupled with his dry sense of humor. He was always good at listening to different sides of an argument and reaching a decision that everyone felt good about."

Rose served from 1951 to 1973 on the faculty of SUNY Buffalo School of Medicine, where he and his mentor Ernest Witebsky discovered in 1956 that Hashimoto's diseasechronic lymphocytic thyroiditis, or hypothyroidismcould be reproduced in experimental animals by immunization with thyroglobulin, which revealed that animals produced an immune response that inflamed and destroyed their own thyroid glands. This landmark discovery of autoimmunity led to the creation of a whole new class of disease and opened the door for research for treatments and cures.

Image caption: Noel R. Rose

He then discovered the genetic basis of autoimmune disease. His lab showed for the first time that the major histocompatibility complex contains the main genes that determine the risk for all autoimmune disease. From 1973 to 1982, he was professor and chair of the Department of Immunology and Microbiology at Wayne State University School of Medicine.

Rose joined Johns Hopkins in 1982 as chair of what was then known as the Department of Immunology and Infectious Diseases and is now the W. Harry Feinstone Department of Molecular Microbiology and Immunology at the Bloomberg School. He held a full-time joint appointment in the Johns Hopkins School of Medicine's Department of Pathology, where he directed the Pathobiology Graduate Program.

After stepping down as chair in 1993, Rose directed the Department of Pathology Division of Immunology for five years and served as president of the Bloomberg School Faculty Senate. He also directed the Johns Hopkins Center for Autoimmune Disease Research, which he founded in 1999.

"As his successor as department chair, I benefitted greatly from Noel's perspective and advice. In his own quiet and considerate way, he contributed to science and the School of Public Health in multiple ways," recalled Diane Griffin, University Distinguished Service Professor at the Bloomberg School who served as chair of Molecular Microbiology and Immunology from 1994 through 2014. "He was a founding contributor to the field of autoimmunity and continued to make pioneering contributions to our understanding of these diseases."

Rose was born in Stamford, Connecticut, on December 3, 1927. His mother was a homemaker and his father a physician. He received his bachelor's degree in zoology in 1948 from Yale University, a PhD in medical microbiology from University of Pennsylvania in 1951, and his MD in 1964 from the State University of New York, Buffalo.

His numerous accolades include the Abbot Award, Professional Recognition Award, and Founder's Distinguished Service Award from the American Society for Microbiology; Ernest Lyman Stebbins Medal from Johns Hopkins Bloomberg School of Public Health, the Bloomberg School's most prestigious award; Nikolaus Copernicus Medal from the Polish Academy of Sciences; Presidential Award from the Clinical Immunology Society, and Golden Goose Award from the American Association for the Advancement of Science.

His prominent national and global leadership roles included director of the Pan American Health Organization/World Health Organization Collaborating Center for Autoimmune Disorders and chair of the Autoimmune Diseases Coordinating Committee at the National Institutes of Health. Rose was editor or co-editor of 23 books, most notably the textbook The Autoimmune Diseases, and published more than 880 articles and chapters in the fields of immunology, immunopathology, autoimmunity, and autoimmune diseases.

In 1991, Rose and Virginia T. Ladd founded the American Autoimmune Related Diseases Association, the primary research and advocacy group for autoimmune diseases. For 20 years, Rose chaired the organization's scientific committee and spearheaded the biannual Noel R. Rose Scientific Colloquium, which convenes the nation's leading researchers in autoimmune and related diseases.

Rose is survived by his wife of 69 years, Deborah H. Rose; children Alison Rose Weinstock, David Richard Rose, Bethany Rose Kramer, and Jonathan Richard Rose; 10 grandchildren; and five great-grandchildren.

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Noel R. Rose, widely regarded as the father of autoimmune disease research, dies at 92 - The Hub at Johns Hopkins

IRVINE, Calif., Aug. 4, 2020 /PRNewswire/ --Seragon Pharmaceuticals, Inc. today announced GenomeScore, the first artificial intelligence platform combining epigenetic and transcriptome analyses to predict a person's aging speed and assess different aspects of physiological function.

"How you age depends on how you lived your life up to this point, and GenomeScore looks into what's currently occurring in your body at the molecular level," said Lead Scientist Aake Vaestermark of Seragon Pharmaceuticals. While traditional genetic tests reveal genetic predisposition, the company's new platform allows individuals to understand how well their biological system is performing over time. "Conventional genetic tests are like comparing the cars that haven't been used, but our new platform is like comparing used cars to see if all the components are up and running after all those years."

Unlike existing epigenetic age tests that only provides an overall body age, GenomeScore's neural network-trained algorithms paint a more detailed and complete picture of the body's age in different areas. The platform shows both the individual's biological age and predicts one's body aging rate. By combining transcriptome analysis, an assessment of gene expression, the platform can also predict the "age" of other physiological functions such as the immune and endocrine systems.

"The platform could act like a warning sign if one of your organs is growing old particularly fast," said Dr. Vaestermark. "It can be a way of finding your weakest point physically, and people want to know that."

Traditionally, people estimate their age through chronological years, the number that one celebrates on their birthday each year. However, individuals age in different paces due to factors like genetics and lifestyles. Based on a set of proprietary epigenetic, metabolic and gene expression markers, GenomeScore platform can predict an individual's speed of aging and physiological changes.

GenomeScore provides a novel way of measuring health parameters that may translate to clinical applications for consumers to better predict how lifestyles and other factors impact their process of aging. For scientists and physicians, a better understanding of how individual ages may provide the opportunity to develop personalized medicine to target patients' medical needs in the future.

Through Seragon Pharmaceuticals' dedication to innovation and technology advancement, the company has developed a set of microarray chips based on the GenomeScore algorithms to make the platform more accessible. The technology further expands the company's bioinformatics research and product portfolio, fulfilling its commitment to creating healthier communities through advancing science and product development.

About Seragon PharmaceuticalsSeragon Pharmaceuticals, Inc., headquartered in Irvine, California, is a research-based biopharmaceutical company dedicated to improving human and animal health through innovative science. Seragon Pharmaceuticals is committed to applying cutting-edge scientific and technological advancements to the fields of metabolism, gene therapy and bioinformatics. From the research end to consumer products and clinical applications, Seragon strives to bring people access to the most significant breakthroughs in medicine. For more information, please visit http://www.seragon.com.

Media Contact: Tiffany Chen [emailprotected]

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https://www.seragon.com

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Seragon Announces GenomeScore, the First AI Platform to Measure Rate of Aging and Physiological Changes - PRNewswire

DURHAM, N.C., July 29, 2020 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that it has acquired exclusive rights to a gene therapy targeting GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina and is a leading cause of blindness in children, from Sanofi, which originally licensed it from the University of Florida. The therapy was created in the laboratory of Atsena Founder and Chief Scientific Officer Shannon Boye, Ph.D., and Founder and Chief Technology Officer Sanford Boye, M.Sc., at the University of Florida.

We are thrilled that our gene therapy for LCA1 is coming home to Atsena and that we will have the opportunity to further its development, said Shannon Boye. Atsena was founded to advance treatments for inherited retinal diseases and believes in centering patients perspectives and needs in all we do. We are honored to continue to work with LCA1 patients and their families as we strive to treat this debilitating disease.

LCA is the most common cause of blindness in children, impacting two to three per 100,000. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this inherited retinal disease.

Atsena has an ongoing Phase I/II clinical trial evaluating this gene therapy in LCA1 patients. The second cohort in the trial is expected to be dosed in the fall of 2020.

Atsenas gene therapy has the potential to be a major advance in treating blindness in both children and adults affected by this inherited retinal disease, said Benjamin Yerxa, Ph.D., Chief Executive Officer of the Foundation Fighting Blindness and Atsena board director. The foundation was instrumental in supporting proof of concept studies in the founders labs over the last 15 years. Now, via investment in Atsena through our Retinal Degeneration (RD) Fund, we are excited to support this potential breakthrough treatment for LCA1.

Atsena closed a Series 1 funding of $8.15 million in April 2020, led by founding investors Hatteras Venture Partners and the Foundation Fighting Blindness RD Fund with participation by Osage University Partners, PBM Capital and the University of Florida. Patrick Ritschel, M.B.A., co-founder and former President of gene therapy company StrideBio, serves as Atsenas Chief Executive Officer.

Atsena is pleased to have the support of an enthusiastic investor base that shares our dedication to bringing the life-changing power of genetic medicine to patients living with LCA1 and other forms of blindness, said Ritschel. We look forward to working closely with our investors and patients as we continue to grow, and expect to announce additional milestones later this year.

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company, focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness. The company has an ongoing Phase I/II clinical trial evaluating a potential therapy for one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome the hurdles presented by inherited retinal disease, and its approach is guided by the specific needs of each patient condition. Founded by pioneers in ocular gene therapy, Atsena has a licensing, research and manufacturing collaboration with the University of Florida and is headquartered in North Carolinas Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

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

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Atsena Therapeutics acquires exclusive rights to Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis - GlobeNewswire