Gene therapy trials for hereditary retinal degenerationsView Session View Presentation
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Abstract Number: 992
AuthorBlock: Jacque L. Duncan1
1Ophthalmology, Univ of California - SF, San Francisco, California, United States;
DisclosureBlock: Jacque L. Duncan, AGTC Code C (Consultant) , Allergan, Inc. Code F (Financial Support) , Editas Medicine, Inc. Code C (Consultant) , Neurotech USA, Inc. Code F (Financial Support) , Nightstar Limited Code F (Financial Support) , ProQR Therapeutics, Inc. Code C (Consultant) , Spark Therpeutics, Inc. Code C (Consultant) , SparingVIsion Code C (Consultant) , Second Sight Medical Products Code F (Financial Support)
With the advent of the first FDA approved gene therapy for eye disease in patients with RPE65-related retinal degeneration, there is increased interest by industry and patients in gene-based therapeutic clinical trials. What are the most promising approaches, and what are the most challenging aspects of gene based therapies? The presentation will provide an overview and update of gene therapy trials for patients with inherited retinal degenerations.