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CANE FOUNDATION Posts

Ophthotech Announces First Patient Enrolled in the Phase 2b Clinical Trial of Zimura®, Complement C5 Inhibitor, for the Treatment of Autosomal Recessive Stargardt Disease (STGD1)

Posted in Technology & Research

Ophthotech Corporation (NASDAQ: OPHT) announced today that the first patient has been enrolled in the Phase 2b randomized, double masked, sham controlled clinical trial assessing the efficacy and safety of Zimura® (avacincaptad pegol), complement C5 inhibitor, in patients with autosomal recessive Stargardt disease (STGD1). STGD1 is an inherited orphan retinal disease causing vision loss during childhood or adolescence. Currently there are no FDA or EMA approved treatment options available for patients with STGD1. Over 30 sites have been identified for this clinical trial, making it one of the largest interventional clinical trials for STGD1 to date. The scientific details of the Stargardt clinical trial will be presented at the 2018 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Honolulu, Hawaii.

AGTC Files Investigational New Drug Application for the Treatment of X-linked Retinitis Pigmentosa Caused by Mutations in the RPGR Gene

Posted in Technology & Research

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Aug. 10, 2017 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has filed an Investigational New Drug application (IND) with the U.S. Food and Drug Administration to conduct a Phase 1/2 clinical trial of the company’s gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.