RetroSense Gene Therapy Drug Gets FDA OK For Human Trials

ANN ARBOR — The pharmaceutical startup RetroSense Therapeutics LLC said Monday that its Investigational New Drug application for its lead drug candidate, RST-001, has received clearance from the United States Food and Drug Administration.

RetroSense is developing RST-001 for the treatment of retinitis pigmentosa, a genetic condition that leads to the progressive degeneration of rod and cone photoreceptors — the cells found in the retina that sense light — resulting in severe vision loss and blindness.

With its IND now in effect, RetroSense expects to initiate a Phase 1 and 2 clinical trial by year-end in order to evaluate the safety and, potentially, effectiveness of RST-001.

In Phase 1 trials, researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase 2 trials, the drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety. And in Phase 3, the drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

RetroSense Therapeutics is developing RST-001 as a first-in-its-class gene therapy application of optogenetics. Optogenetics refers 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 working to confer new light sensitivity to the retina, with the expectation of some degree of improved or restored vision for affected patients.

In 2014, the FDA granted Orphan Drug designation for RST-001 based on its development as a treatment of RP, a rare disease that affects an estimated 100,000 people in the United States. As a designated Orphan Drug, RST-001 is eligible for various development incentives under the Orphan Drug Act, including a potential waiver from FDA’s application user fees, tax incentives and Orphan Drug exclusivity.

“The IND for RST-001 is an important milestone for the company,” RetroSense CEO Sean Ainsworth said. “This brings us one step closer to realizing our ambition of improving vision in those individuals with currently incurable blindness. There is great promise for the clinical application of optogenetics and this first human clinical trial should provide key insights into the potential for this therapy to treat diseases affecting the eye or brain.”

Dr. Zhuo-Hua Pan, inventor of the company’s optogenetic approach, added: “My hope from early on was to see our work improve the lives of people with vision defects. It is great to see the approach moving imminently into human clinical studies.”

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