A natural history study shows patients who have Leber’s hereditary optic neuropathy with the ND4 mutation have a dramatic and irreversible decline in visual acuity, a press release from GenSight Biologics said.
The retrospective, cross-sectional REALITY observational study highlights the poor prognosis LHON patients have without treatment, the release said.
In contrast, subjects in the phase 3 REVERSE and RESCUE trials, which treat LHON patients with GenSight’s adeno-associated virus vector gene therapy Lumevoq, have shown visual acuity improvement.
“These findings highlight just how remarkable the visual trends in REVERSE and RESCUE are,” Bernard Gilly, co-founder and CEO of GenSight, said in the release. “The findings will support our effort to provide regulatory authorities with the most robust insights about the natural history of the disease, so that we can convincingly demonstrate the therapeutic benefit delivered by Lumevoq.”