Beacon gene therapy meets main goal in XLRP trial
Beacon Therapeutics reported that its gene therapy laru-zova met the primary endpoint in a pivotal late-stage trial for X-linked retinitis pigmentosa (XLRP). In 85 male patients aged 12 to 48, 24% on a low dose and 31% on a high dose gained 15 or more letters on a low-light visual acuity test, while control patients achieved no such gains. Adverse events were mostly mild to moderate, with two serious events linked to the surgical delivery. Beacon will begin a rolling approval submission by year end and will present fuller results at a medical meeting in October.
A one-time gene therapy showed significant low-light vision gains in XLRP.
Context
X-linked retinitis pigmentosa causes progressive vision loss and often affects boys. Other gene therapy programs for XLRP had mixed results, including past failures. Beacon ran a pivotal trial that met its main goal and will seek…
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