PRVL - Dosing underway in Prevail Therapeutics' gene therapy study for a type of dementia

Prevail Therapeutics (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 investiganational 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.Shares rise 1.4% in premarket

For further details see:

Dosing underway in Prevail Therapeutics' gene therapy study for a type of dementia