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Sustained Therapeutic delivery for the eye

Clinical Advisory Board

Ian J. Constable, M.D.

Dr. Constable is Founder and Professor of Ophthalmology at the Lions Eye Institute, University of Western Australia, and is an expert in gene therapy approaches to retinal disease.

Mark S. Blumenkranz, M.D.

Dr. Blumenkranz is Professor and Chairman at the Byers Eye Institute at Stanford. He is a renowned expert in retinal disease and has founded several Silicon Valley startup companies.

Steven D. Schwartz, M.D.

Dr. Schwartz is Professor of Ophthalmology and Chief of Retina Division at the UCLA Jules Stein Eye Institute. He is a renowned expert in retinal diseases and early-stage clinical research.

Judy Gordon, D.V.M.

Dr. Gordon is a Clinical Regulatory Consultant with extensive experience in Ophthalmology products.

Steven Butler, Ph.D.

Dr. Butler is a Consulting Biostatistician. As an Associate Director at Genentech, he served as the lead biostatistician and development sub-team leader for Lucentis.

The results of a Phase 1, 52-week trial of Avalanche Biotechnologies’ rAAV.sFLT-1 (AVA-101) gene therapy for the treatment of wet age-related macular degeneration (wAMD) have been published online by The Lancet. The study sought to evaluate the safety of rAAV.sFLT-1 via a single subretinal injection. Results of the study demonstrate that rAAV.sFLT-1 was safe and well tolerated, and support ocular gene therapy as a potential long-term treatment option for wAMD.

“The use of gene therapy to reprogramme genetically normal cells of the retina to take on additional functions opens a new chapter in potential AAV applications.”
– Dr. Robert MacLaren, Moorsfield Eye Hospital

The complete article and editorial commentary by Dr. MacLaren, are available from The Lancet.

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