Prevail Therapeutics Receives IND Approval for Gene Therapy in Patients with Frontotemporal Dementia with GRN Mutation

Prevail Therapeutics Receives IND Approval for Gene Therapy in Patients with Frontotemporal Dementia with GRN Mutation

Prevail Therapeutics announced that the U.S. FDA has approved an Investigational New Drug (IND) application for its experimental gene therapy program, PR006, for the treatment of frontotemporal dementia patients with GRN mutation (FTD-GRN).

With this approval, the company plans to advance the PROCLAIM Phase 1/2 clinical trial to investigate the safety and tolerability of PR006, as well as measure key biomarkers and exploratory efficacy endpoints in patients with FTD-GRN. The Company plans to begin dosing for PROCLAIM this year.

The FDA has previously granted Orphan Drug Designation for PR006 for the treatment of patients with FTD.

About PR006

PR006 is being developed as a potential one-time gene therapy for FTD-GRN, a progressive neurodegenerative disease caused by mutations in the GRN gene 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 a GRN Mutation

Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimer’s disease. FTD affects 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union. Frontotemporal dementia with a GRN mutation (FTD-GRN) represents 5-10% of all patients with FTD. FTD results 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 for FTD or FTD-GRN.