Authorization

Prevail Therapeutics Granted Composition of Matter Patent for Experimental Gene Therapy Program PR001

NEW YORK, Nov. 18, 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) onANovember 17, 2020Aissued a composition of matter patent, U.S. Patent No. 10,837,028,Awith claims directed to the AAV vector used in PR001, Prevaila??s experimental gene therapy program for the treatment of Parkinsona??s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). The base patent term extends until October 3, 2038, excluding patent term extensions or coverage in additional related patent filings.
a??We are excited to make important progress this year with PR001, which is being evaluated in the Phase 1/2 PROPEL trial for patients with Parkinsona??s disease with GBA1 mutations and in the Phase 1/2 PROVIDE trial for patients with Type 2 Gaucher disease,a?? said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. a??We are advancing clinical development of PR001 to make a potentially transformative difference for these patients who currently have no approved treatment options.a??The Company recently announced that patient dosing has continued in the Phase 1/2 PROPEL clinical trial of PR001 for PD-GBA patients, and it expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021. The Company expects to initiate enrollment of the Phase 1/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease patients in the fourth quarter of 2020 and currently anticipates it will provide the next update on PR001 biomarker and safety data for nGD in 2021.The U.S. Food and Drug Administration has granted Fast Track designations for PR001 for the treatment of PD-GBA and nGD. In addition, the FDA granted PR001 Rare Pediatric DiseaseAdesignation for the treatment of nGD, and Orphan DrugAdesignation for the treatment of patients with Gaucher disease.About Prevail Therapeutics
Prevail 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. The Company is developing PR001 for patients with Parkinsona??s disease with GBA1Amutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia withAGRNAmutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.
See also:
Leave a comment
News
  • Latest
  • Read
  • Commented
Calendar Content
«     2021    »
 123
45678910
11121314151617
18192021222324
25262728293031