Ann Arbor Firm Gets Orphan Drug Status For RP Treatment

ANN ARBOR — Ann Arbor-based RetroSense Therapeutics LLC said it had received “orphan drug” status from the federal Food and Drug Administration for its lead drug candidate, RST-001, intended to treat the eye disease retinitis pigmentosa.

RetroSense Therapeutics is developing RST-001 as a first-ever gene therapy application of optogenetics designed to restore vision to those affected by RP. Optogenetics is a term for conferring light sensitivity to cells that were not previously light sensitive. By applying optogenetics to retinas in which the eye’s “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, no matter which gene or mutation has caused it.

The drug 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.

Said Sean Ainsworth, RetroSense 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 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, tax credits for clinical testing expenses conducted after orphan designation is received, and reduced regulatory fees.

Retinitis pigmentosa is a genetic condition which leads to the progressive degeneration of rod and cone photoreceptors, the cells 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.

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