RetroSense Therapeutics Granted Orphan Drug Designation for Lead Product RST-001 for Retinitis Pigmentosa

Unique approach combines gene therapy and optogenetics with the intention to treat blindness

ANN ARBOR, Mich.--()--RetroSense Therapeutics, LLC, a privately-held biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for the Company’s lead product RST-001 for the treatment of retinitis pigmentosa (RP). RetroSense Therapeutics is developing RST-001 as a first-in-class gene therapy application of optogenetics designed to restore vision to those affected by RP.

Optogenetics refers broadly to means of conferring light sensitivity to cells that were not previously, or natively light sensitive. By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, RetroSense is conferring new light sensitivity to the retina, with the expectation of improved or restored vision. RST-001 is expected to have application to all forms of RP, independent of causative gene or mutation.

The Company's approach to using optogenetics in vision restoration is based on pioneering, proprietary research conducted at Wayne State University’s Kresge Eye Institute and Department of Anatomy and Cell Biology, and Massachusetts General Hospital. RetroSense has worldwide exclusive rights to the relevant intellectual property from both institutions.

“We are pleased that the FDA has granted Orphan Drug status to our lead product, RST-001,” stated Sean Ainsworth, RetroSense Therapeutics’ CEO. “This significant milestone will enable us to continue to develop new and innovative treatments for retinitis pigmentosa, a truly debilitating condition. We are hopeful that the benefits associated with Orphan Drug status will better enable us to advance RST-001 through development and ultimately into the marketplace where it may benefit many who are suffering from blindness due to retinitis pigmentosa.”

The FDA Office of Orphan Products Development supports the evaluation and development of products that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions. The FDA awards Orphan Drug designation as an incentive to develop drugs and biological therapeutics for diseases that affect fewer than 200,000 people in the Unites States. The benefits of Orphan Drug designation include a seven year period of market exclusivity following FDA approval, certain tax credits for clinical testing expenses conducted after orphan designation is received, and reduced regulatory fees.

About Retinitis Pigmentosa

Retinitis pigmentosa is a genetic condition which leads to the progressive degeneration of rod and cone photoreceptors (cells found in the retina of the eye that sense light). Loss of these cells results in severe vision loss and blindness. There are currently no FDA-approved drugs to treat RP.

About RetroSense Therapeutics

RetroSense Therapeutics is a privately-held biotechnology company developing life-enhancing gene therapies designed to restore vision in patients suffering from blindness due to retinitis pigmentosa (RP) and advanced dry age-related macular degeneration (advanced dry-AMD). There are currently no FDA approved drugs to improve or restore vision in patients with these retinal degenerative conditions. RetroSense is led by a team of seasoned veterans with deep experience in taking products from the discovery stage through to the clinic. For more information about RetroSense, visit http://www.retro-sense.com/.

Contacts

RetroSense Therapeutics, LLC
Amy Caterina, 734-926-9314
Media Relations
amy@retro-sense.com

Release Summary

RetroSense Therapeutics Granted Orphan Drug Designation for Lead Product RST-001 for Retinitis Pigmentosa. Unique approach combines gene therapy and optogenetics with the intention to treat blindness.

Contacts

RetroSense Therapeutics, LLC
Amy Caterina, 734-926-9314
Media Relations
amy@retro-sense.com