Gene Therapy for Leber Congenital Amaurosis (RPE65 Mutations): Applied Genetic Technologies Corporation (AGTC)


Description: This Phase I/II clinical trial, made possible by preclinical studies funded by the Foundation, is evaluating the safety of gene replacement therapy for people with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65. Efficacy is being evaluated, as well. Investigators plan to treat as many as 12 individuals including children and adults. Participants receiving a single subretinal injection of the treatment in one eye. The treatment consists of a corrective RPE65 gene, which is delivered to photoreceptors in the retina by an adeno-associated virus (AAV), a therapeutic man-made virus. The study is being conducted at Oregon Health and Science University (Portland, Oregon).

Trial Status: Ongoing; recruiting participants.

A Vision for Gene Therapy

Additional Information:
AGTC gene therapy clinical trial listing on