Gene Therapy for Leber Congenital Amaurosis (RPE65 Mutations): Hadassah Medical Organization (Jerusalem, Israel)


Description: This Phase I 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 10 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 in Jerusalem, Israel.

Trial Status:
Recruiting participants

Partial List of Participation Criteria:

  1. Diagnosis of Leber congenital amaurosis caused by RPE65 mutation
  2. 8 years of age or older

Clinical Contact:
Devora Marks Ohana
Phone: 00 972 2 6776324
Hadassah Medical Organization (Jerusalem, Israel)

Additional Information:
Hadassah gene therapy clinical trial listing on