Research & Development
Gyroscope Therapeutics Granted FDA Fast Track Designation for GT005, an Investigational Gene Therapy for Dry Age-Related Macular Degeneration
22 September 2020 - - The US Food and Drug Administration has granted Fast Track designation to GT005 for the treatment of geographic atrophy secondary to dry age-related macular degeneration, UK-based clinical-stage retinal gene therapy company Gyroscope Therapeutics Ltd said.

GT005 is an investigational one-time AAV-based gene therapy that is delivered under the retina and is intended to slow the progression of GA that can lead to blindness.

Fast Track designation was granted to GT005 for the treatment of people with GA who have specific mutations in their Complement Factor I gene and low levels of the CFI protein in their blood.

Enrolment in the Phase II EXPLORE study [NCT04437368] to evaluate GT005 in this group of people is underway, the company said.

In addition to EXPLORE, Gyroscope also plans to initiate a second Phase II trial in 2020 that will evaluate GT005 in a broader group of people with GA.

The FDA's Fast Track programme streamlines the review of drugs for serious conditions without FDA-approved treatment options available.

Fast Track designation gives applicants access to more frequent communication with the FDA throughout the review process, and the potential to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete.

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing and delivering gene therapy beyond rare disease to treat a leading cause of blindness, dry AMD.

Its lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina.