The Food and Drug Administration (FDA) has granted Fast Track designation to PR006 (Prevail Therapeutics), an experimental gene therapy, to slow the progression of frontotemporal dementia with a GRN mutation (FTD-GRN).

FTD-GRN is a progressive neurodegenerative disease caused by mutations in the GRN gene. Patients with a GRN mutation have reduced levels of progranulin, a protein critical for lysosomal function, neuronal survival, and normal microglial activities. PR006 delivers a healthy GRN gene using an AAV9 vector to increase progranulin levels in these patients.

The Food and Drug Administration (FDA) recently accepted the Investigational New Drug application for PR006, allowing the Company to proceed with a phase 1/2 trial. 

“FTD-GRN progresses rapidly and there are currently no therapeutic options available,” said Asa Abeliovich, MD, PhD, Founder and CEO of Prevail. “We believe PR006 has the potential to fill this unmet medical need and make a significant impact for patients.”

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The FDA previously granted Orphan Drug designation to PR006 for the treatment of patients with frontotemporal dementia.

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