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Getting the latest healthcare news for you
The FDA has accepted a biologics license application (BLA) for Mogenry, a one-time intravitreal optogenetic gene therapy developed by Nanoscope Therapeutics for patients with severe vision loss due to retinitis pigmentosa. If approved, it would be the first gene-agnostic therapy of its kind. Clinical trial data showed meaningful visual acuity improvements at both 52 and 76 weeks, with a strong safety profile.
The FDA has officially accepted a biologics license application (BLA) for Mogenry (sonpiretigene isteparvovec), Nanoscope Therapeutics' investigational one-time intravitreal optogenetic gene therapy for retinitis pigmentosa (RP). If approved, Mogenry would be the first gene-agnostic therapy to restore vision in patients with severe vision loss from RP — a condition that currently has no approved treatments.
The BLA submission was backed by data from a phase 1/2a trial, the phase 2b/3 RESTORE study, and the long-term REMAIN follow-up trial. Mogenry demonstrated visual acuity improvements at both 52 and 76 weeks in the RESTORE trial, with no treatment-related serious adverse events reported.
Key Takeaways:
Why it matters: Retinitis pigmentosa affects roughly 1 in 4,000 people worldwide and currently has no approved therapies. FDA approval of Mogenry could offer a transformative, one-time treatment option to a patient population with significant unmet need.
