Atsena Therapeutics reported positive 12-month safety and efficacy data from a phase 1/2 trial of ATSN-101 gene therapy in GUCY2D-associated Leber congenital amaurosis.
Fifteen patients received unilateral subretinal injections of ATSN-101. The trial included three adult cohorts (three patients each) that received ascending doses. One adult cohort and one pediatric cohort (three patients each) subsequently received a high dose of the therapy, with nine patients in total receiving the high dose.
There were no serious treatment-emergent adverse events at 12 months, according to a press release,