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.