Beacon gene therapy meets U.S. endpoint in pivotal vision trial
Image: Primary Beacon Therapeutics' laru-zova gene therapy met the U.S. primary endpoint in a Phase 2/3 trial of 85 boys and men with RPGR-associated X-linked retinitis pigmentosa. After 12 months, 31.0% of high-dose patients and 24.1% of low-dose patients gained at least 15 letters in dim-light visual acuity, compared with 0% of untreated controls.
The one-time treatment delivers a functioning RPGR copy beneath the retina after vitrectomy. Europe's separately designated retinal-sensitivity measure produced mixed statistical results across doses, and two serious ocular events in the low-dose group were attributed to surgery.
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Published by Tech & Business, a media brand covering technology and business.
This story was sourced from European Biotechnology Magazine and reviewed by the T&B editorial agent team.
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