1. Sep 23, 2020

AMD gene therapy gets FDA fast track designation

The U.S. Food and Drug Administration (FDA) has granted fast track designation to the GT005 (Gyroscope Therapeutics Limited), an investigational AAV-based gene therapy, for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), according to a company press release.

The designation was granted for patients with specific mutations in their Complement Factor I (CFI) gene and low levels of the CFI protein in their blood.  

“Dry AMD is a life-altering diagnosis and there are currently no FDA-approved medicines available. Research suggests people with dry AMD who have certain CFI mutations that correlate with low CFI levels in the blood have a higher risk of developing AMD,1” said Nadia Waheed, MD, MPH, Chief Medical Officer, in a press release. “We are pleased to receive Fast Track designation for our investigational gene therapy for this high-risk group. We look forward to working with the FDA as we advance our clinical program evaluating the safety and effectiveness of GT005.”

A Phase II study [NCT04437368] on GT005 is currently enrolling patients.

Read the full press release here.