Beacon Therapeutics reported that its X-linked retinitis pigmentosa gene therapy laruparetigene zovaparvovec (laru-zova) met the primary endpoint in the FDA-endorsed Phase II/III VISTA trial, using low-luminance visual acuity (LLVA) as the efficacy measure. The company said responder rates (gain of at least 15 letters at 12 months) improved in both high- and low-dose cohorts versus the untreated control group. The pivotal win moves the program closer to a biologics license application and could further validate gene therapy approaches for inherited retinal disease—where therapeutic windows and outcome measures often prove difficult to align across trials. Beacon’s next steps center on preparing regulatory submissions following the Phase II/III results.
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