Bilateral visual acuity gains reported in phase 3 trial of LHON gene therapy treatment

SAN FRANCISCO — A gene therapy treatment candidate for Leber hereditary optic neuropathy bilaterally improved best corrected visual acuity during a phase 3 trial, according to a speaker here.
“We saw bilateral improvement in both the treated and the sham eyes,” Patrick Yu-Wai-Man, FRCOphth, MBBS, PhD, said at the American Academy of Ophthalmology annual meeting.
Yu-Wai-Man presented the 96-week data from the phase 3 RESCUE trial, which included 39 patients with Leber hereditary optic neuropathy (LHON) treated with either GS010 (rAAV2/2-ND4) from GenSight Biologics or sham. The

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