FDA to consider gene therapy for achromatopsia

Applied Genetic Technologies Corporation has filed a new drug application with the FDA to conduct a clinical trial of a gene therapy product to treat achromatopsia.

The company intends to initiate multi-center clinical trials for their adeno-associated virus (AAV)-gene therapy product targeting the CNGB3 mutation, the most common mutation associated with achromatopsia. AGTC also recently entered into a collaboration with Biogen, allowing the Cambridge-based company to use AGTC’s proprietary technology to make AAV vectors.

"In addition to finalizing our collaboration agreement with Biogen, initiating our phase 1/2 clinical trial for x-Linked retinoschisis, and expanding our product pipeline, we remain on track to have a second product candidate in clinic shortly, pending the FDA's acceptance of the investigational new drug application and approval from the sites' Institutional Review Boards," said Sue Washer, AGTC president and CEO .