Date: 2016-08-10
Type of information: Treatment of the first patient
phase: 1-2
Announcement: treatment of the first patient
Company: RetroSense Therapeutics (USA - MA)
Product: RST-001
Action
mechanism: gene therapy. RST-001 is a first-in-class gene therapy application of optogenetics designed to restore vision to those affected by retinitis pigmentosa. 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 working to confer new light sensitivity to the retina, with the expectation of some degree of improved or restored vision for affected patients. RST-001 is genotype independent, and is expected to have application irrespective of which genetic defect causes the photoreceptors to degenerate. 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.
Disease: retinitis pigmentosa
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Country: USA
Trial
details: The study is composed of two parts. The initial dose-ranging study will examine three dose levels of RST-001 in three separate groups of adult patients with advanced disease. This first part of the study is aimed at determining a single dose of the experimental agent that is safe and well tolerated, to further evaluate in a fourth group of patients. The second part of the study is aimed at obtaining additional safety data at the highest tolerated dose and providing important additional clinical data to guide the design of future efficacy studies. (NCT02556736)
Latest
news: * On August 10, 2016, RetroSense Therapeutics announced that the low dose cohort of patients has been safely dosed in the initial clinical trial to evaluate the safety of RST-001. No inflammation or ocular adverse events were seen in any of these patients, and biological activity has been confirmed. The study is titled Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Uniocular Intravitreal RST-001 in Patients With Retinitis Pigmentosa. * On August 24, 2015, RetroSense Therapeutics announced the Company’s Investigational New Drug (IND) application for RST-001 received clearance from the FDA. RetroSense is developing RST-001 for the treatment of retinitis pigmentosa (RP), a genetic condition that 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 its IND now in effect, RetroSense expects to initiate a Phase I/II clinical trial by year-end in order to evaluate the safety and, potentially, efficacy of RST-001.
