Gene Therapy for Choroideremia: University of Oxford (U.K.)

Description: This 24-month, Phase I/II clinical trial, made possible through preclinical studies funded by the Foundation, is evaluating the safety and efficacy of gene replacement therapy for people with choroideremia. Initially, 12 participants are being recruited for the study. The treatment consists of a corrective REP-1 gene, which is delivered to photoreceptors in the retina by an adeno-associated virus (AAV), a therapeutic man-made virus. Two dose levels of the treatment are being evaluated.

Trial Status: Recruiting participants.

Partial List of Participation Criteria:

  1. Diagnosis of choroideremia
  2. Males, 18 years of age or older

Clinical Contacts:

  • Robert E MacLaren, M.B., Ch.B. D.Phil., Study Chair
  • University of Oxford, Oxford Radcliffe Hospitals NHS Trust and Moorfields Eye Hospital


Additional Information: Choroideremia Gene Therapy Clinical Trial Listing on