Prevail Therapeutics starts gene therapy study for type of dementia

 

• Prevail Therapeutics (NASDAQ:PRVL) initiates dosing in Phase 1/2 PROCLAIM trial evaluating PR006, an investigational AAV9 gene therapy delivering the GRN gene, for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN).
• The trial with up to 15 subjects will investigate the safety and tolerability of PR006 as well as key biomarkers and exploratory efficacy endpoints. Biomarker and safety analysis on a subset of patients enrolled in the trial is expected in 2021.
• The investigational gene therapy has been granted US & Europe Orphan Drug designation for FTD and Fast Track designation for FTD-GRN.
• PR006 is designed to increase levels of a protein called progranulin in the brains of FTD-GRN patients by delivering a healthy GRN gene via an AAV9 vector. Progranulin, up-regulated in a range of neuroinflammatory conditions, is believed to play a key role in FTD.
• https://seekingalpha.com/news/3643647-dosing-underway-in-prevail-therapeutics-gene-therapy-study-for-type-of-dementia