Gene Therapy for Leber Congenital Amaurosis (RPE65 Mutations): Moorfields Eye Hospital (London, UK)


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 have treated 10 individuals including children and adults. Participants are 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 treatment has restored significant vision for some participants (see articles below).

Trial Status: Ongoing, recruiting participants.

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Partial List of Participation Criteria:

  1. Diagnosis of Leber congenital amaurosis caused by RPE65 mutation
  2. 5 to 30 years of age

Clinical Contact:
James Bainbridge, PhD FRCOphth
Phone: 02076084023
Moorfields Eye Hospital NHS Foundation Trust (London, England)

Additional Information:
Moorfields gene therapy clinical trial listing on