Gene Therapy for Stargardt Disease (ABCA4 Mutations): Oxford Biomedica


Description: Oxford BioMedica, a gene therapy company in the U.K., has launched a Phase I/IIa clinical trial of its StarGen™ gene therapy for people with Stargardt disease. The treatment employs the company’s proprietary LentiVector® platform technology for the delivery of a corrective ABCA4 gene to the retina. The company is now conducting the trial at Oregon Health and Science University (Portland, Oregon), and is planning to begin a human study soon at Centre Hospitalier Nationale D’Opthalmologie des Quinze-Vingts (Paris, France). As many as 28 participants will be enrolled in the study. Three dose levels of the treatment will be evaluated for safety, tolerability and biological activity. The clinical trial was made possible by preclinical research funded by the Foundation Fighting Blindness.

Trial Status: Recruiting

Partial List of Participation Criteria:

  1. 18 years of age or older
  2. Diagnosis of Stargardt disease

Clinical Trial Contact:

David Wilson, M.D.
(503) 494-3795
Casey Eye Institute, Oregon health & Science University
Portland, Oregon 97239

Jose Sahel, M.D., Ph.D,
Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts
Paris, France, 75571

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Additional Information:
StarGen clinical trial listing on