First-of-Kind Stem Cell-Based Tx Being Tested for Retinitis Pigmentosa

Scientists from University of California Irvine’s Gavin Herbert Eye Institute and Sue & Bill Gross Stem Cell Research Center announced the start of patient enrollment for the first clinical trial evaluating the use of retinal progenitor cells to treat retinitis pigmentosa.

The Phase 1/2a open-label trial is aiming to study the safety of retinal progenitor cells injected into the vitreous cavity at two different doses in patients with late-stage retinitis pigmentosa. The study will enroll 16 patients to receive a single injection into the worst-seeing eye under topical anesthesia; to date, four patients with retinitis pigmentosa have received cell injections. Patients will be followed for 12 months with monitoring of safety and efficacy parameters.

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The first-of-kind stem cell-based treatment being studied is intended to preserve vision by exerting its effect when degenerating photoreceptors in the patient’s retina can be protected and possibly reactivated–a less of a challenge than replacing lost cells.

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