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News 1/25/2018

James Wilson, MD, PhD, from the University of Pennsylvania, Joins the Rett Syndrome Research Trust Gene Therapy Consortium

The Rett Syndrome Research Trust (RSRT) is pleased to announce that James Wilson, MD, PhD, director of the University of Pennsylvania Gene Therapy Program and the Rose H. Weiss Orphan Disease Center, has joined our Gene Therapy Consortium.

News 12/19/2017

FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss

The U.S. Food and Drug Administration today approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

News 11/06/2017

FDA Clears IND Application For GALGT2 Gene Therapy For Duchenne Muscular Dystrophy

The FDA recently cleared an investigational new drug (IND) application for the GALGT2 gene therapy program for Duchenne muscular dystrophy (DMD) filed by Sarepta Therapeutics and Nationwide Children’s Hospital. Following the FDA clearance, Nationwide Children’s will continue to advance the program by initiating a Phase 1/2a clinical trial in individuals with DMD by the end of 2017 to assess gene expression after gene therapy with GALGT2.

News 8/30/2017

FDA approval brings first gene therapy to the United States

The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.