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Luxturna delivers an adeno-associated virus carrying the correct RPE65 gene into the retina.
By Ava Kikut

Scheie Vision Annual Report 2017*

On December 19, 2017, the Food and Drug Administration (FDA) approved Luxturna, a gene therapy for certain forms of inherited blindness. This decision was informed by a unanimous endorsement (16-0 vote) by the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee on October 12. Luxturna is the first gene therapy for an inherited disease available in the United States. The drug's approval marks the beginning of a new era for the treatment of blinding conditions and gene therapy research.

Luxturna targets mutations of the RPE65 gene, which are associated with a variety of vision-impairing retinal disorders, including Leber Congenital Amaurosis (LCA) and retinitis pigmentosa. Administered through an injection, Luxturna delivers an adeno-associated virus (AAV) carrying the correct RPE65 gene into the retina, restoring photoreceptor responses within 30 days. The treatment, which was developed in collaboration with Spark Therapeutics, has already enabled patients in clinical trials to gain and maintain vision. Luxturna is the product of 25 years of rigorous research, led by Jean Bennett, MD, PhD, and Al Maguire, MD, who established the Center for Advanced Retinal and Ocular Therapeutics (CAROT) in 2014. Currently, more than 265 genes are known to cause inherited retinal disease. The pioneering work at CAROT has the potential to not only transform the lives of individuals with RPE65 mutations, but to lead to treatments for millions of individuals now facing a life of blindness.

The FDA endorsement and approval has made headlines across several major news outlets, including NBC, CNN, CBS, ABC, FOX, NPR, The New York Times, Washington Post, USA TODAY, San Francisco Chronicle, The Bulletin, Forbes Magazine, MIT Technology Review, HealthDay, and the Philadelphia Inquirer. Drs. Bennett and Maguire were also featured in a PBS special, The Gene Doctors. For more exciting details, see the press release from Penn Medicine.

 *Updated 12/19/2017

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