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news.RetroSense Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its investigational new drug (IND) application for RST-001, a gene therapy candidate, being developed to treat retinitis pigmentosa (RP), a genetic condition.
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Retinitis pigmentosa leads to the progressive degeneration of rod and cone photoreceptors (cells found in the retina that sense light), resulting in severe vision loss and blindness.
With the IND now in effect, the company intends to start a Phase I/II trial by year-end in order to evaluate the safety and, potentially, efficacy of RST-001.
The company is developing RST-001 as a first-in-class gene therapy application of optogenetics, which 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, the company is working to confer new light sensitivity to the retina, expecting some degree of improved or restored vision for affected patients.
RetroSense Therapeutics CEO Sean Ainsworth said: "The IND for RST-001 is an important milestone for the company. 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."
Last year, 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 US.