Prevail Therapeutics Granted Composition of Matter Patent for Experimental Gene Therapy Program PR006 – GlobeNewswire

NEW YORK, July 27, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc.(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) on June 23, 2020 issued a composition of matter patent, U.S.PatentNo.10,689,625,with claims directed to the AAV vector used in PR006, Prevails experimental gene therapy program for the treatment of frontotemporal dementia patients withGRNmutations (FTD-GRN). The base patent term extends until October 2038, excluding patent term extensions or coverage in additional related patent filings.

At Prevail, we are working continuously to bring innovative treatments to patients with neurodegenerative diseases, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. FTD-GRN is a devastating condition, with no disease-modifying therapeutic options available. We are excited about the possibility of making a significant impact with PR006.

In March of this year, Prevail announced an active IND for the Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN. TheU.S. Food and Drug Administration(FDA) has granted the therapy Fast Track Designation.

PR006 is being developed as a potential one-time gene therapy for FTD-GRN, a progressive neurodegenerative disease caused by mutations in theGRNgene that reduce production of progranulin, a protein critical for lysosomal function, neuronal survival and normal microglial activities. The progranulin deficiency leads to lysosomal dysfunction, ineffective protein degradation and recycling, neuroinflammation, and ultimately neurodegeneration and death, typically within three to ten years of diagnosis.

PR006 is designed to increase progranulin levels in the brains of FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

About Frontotemporal Dementia with GRNMutationsFrontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimers disease. FTD affects 50,000 to 60,000 people in theU.S.and 80,000 to 110,000 individuals in theEuropean Union. FTD-GRN represents 5-10% of all patients withFTD.FTDresults from the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language.In FTD-GRN patients, reduced levels of progranulin lead to age-dependent lysosomal dysfunction, neuroinflammation, and neurodegeneration. There are no approved treatments forFTDor FTD-GRN.

AboutPrevail TherapeuticsPrevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. In addition to PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN), the company is developing PR001 for patients with Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease, and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort withThe Silverstein Foundationfor Parkinsons with GBA and OrbiMed, and is headquartered inNew York, NY.

Forward-Looking Statements Related to PrevailStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevails ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevails filings with theSecurities and Exchange Commission(SEC), including the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the period endedMarch 31, 2020, filed with theSEConMay 14, 2020, and its other documents subsequently filed with or furnished to theSEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Mary CarmichaelTenBridge Communicationsmary@tenbridgecommunications.com617-413-3543

Investor Contact:investors@prevailtherapeutics.com

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Prevail Therapeutics Granted Composition of Matter Patent for Experimental Gene Therapy Program PR006 - GlobeNewswire

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