Visual acuity gains reported in phase 3 trial of Leber hereditary optic neuropathy treatment

GenSight Biologics’ gene therapy treatment candidate for Leber hereditary optic neuropathy showed continuous recovery of visual function in a phase 3 trial, according to a press release.
Best corrected visual acuity improved by an equivalent of 21 ETDRS letter from nadir between weeks 48 and 72 in eyes treated with GS010 (rAAV2/2-ND4) in the 39-patient RESCUE trial.
Sham-treated eyes has a BCVA evolution of 21.7 ETDRS letters, so a statistically significant difference in visual acuity between GS010 and sham-treated eyes could not be shown, the release said.
“We are excited and

Full Story →