FDA grants fast track designation to gene therapy candidate for achromatopsia

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The FDA has granted fast track designation to AAV-CNGA3, a gene therapy candidate for the treatment of achromatopsia, according to a press release from MeiraGTx.

AAV-CNGA3, designed to restore cone receptor function via subretinal injection, is currently undergoing a phase 1/2 clinical trial in patients with achromatopsia (ACHM) due to CNGA3 gene mutations.

“We are very pleased to have received fast track designation for AAV-CNGA3 and that the FDA has recognized a significant need exists to quickly advance new therapies for those with ACHM,” Alexandria Forbes, PhD, president and CEO of MeiraGTx, said in the release. “ACHM is a serious and debilitating disease, and we look forward to communicating closely with the FDA as we continue the clinical advancement of AAV-CNGA3.”

AAV-CNGA3 previously received orphan drug designation from the FDA and European Medicines Agency, as well as rare pediatric disease designation from the FDA.