Genzyme's Gene Therapy for the Treatment of Wet AMD


Genzyme is conducting a Phase I clinical trial of gene therapy for the treatment of wet age-related macular degeneration (AMD). The treatment involves the delivery of a gene that produces a protein which halts the production of vascular endothelial growth factor (VEGF). VEGF is a protein that leads to the proliferation of leaky blood vessels underneath the retina that causes vision loss in wet AMD. An adeno-associated virus is used for gene delivery. As many as 34 participants will be enrolled in the study. Four doses of the treatment are being evaluated.

Trial Status: Recruiting participants.

Partial List of Participation Criteria:

  1. 50 years of age or older
  2. Diagnosis of wet age-related macular degeneration

Clinical Contact:
(800) 745-4447
(617) 252-7832
Refer to identifier NCT01024998

Recruiting trial locations include Retinal Consultants of Arizona (Phoenix), Johns Hopkins University School of Medicine (Baltimore), and Ophthalmic Consultants of Boston.

Additional Information:
Genzyme’s gene therapy clinical trial listing on