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FDA Grants Orphan Drug Designation for RetroSense Therapeutics' Optogenetics Gene Therapy for Retinitis Pigmentosa

The US Food and Drug Administration (FDA) has granted Orphan Drug designation for the RetroSense Therapeutics' RST-001, a first-in-class gene therapy application of optogenetics designed to restore vision to those affected by retinitis pigmentosa (RP).

Optogenetics refers broadly to a means of conferring light sensitivity to cells that were not previously or natively light sensitive. It is based on research conducted at Wayne State University's Kresge Eye Institute and Department of Anatomy and Cell Biology, as well as Massachusetts General Hospital.

Applying optogenetics to these retinas with degenerated rod and cone photoreceptors is done to improve or restore vision. RST-001 is expected to be used to treat all forms of RP, independent of causative gene or mutation.

The FDA Office of Orphan Products Development supports the evaluation and development of products intended for safe and effective treatment, diagnosis, or prevention of rare diseases or conditions. The Orphan Drug designation is an incentive to develop drugs and biological therapeutics for diseases that affect fewer than 200,000 people in the US.

Updated November 5, 2014