In a phase 1/2 trial, researchers tested ATSN-101, a gene therapy for Leber congenital amaurosis 1, a rare genetic disorder that typically causes blindness in early childhood.
Fifteen patients with mutations in the GUCY2D gene were treated with increasing doses of ATSN-101. Overall, the subretinal therapy was well tolerated, with most side effects related to the surgery rather than the drug.
The trial also showed patients who received the highest dose of the gene therapy made progress in night vision and overall blindness.
To the researchers' knowledge, gene therapy ATSN-101 is currently the only gene therapy targeting LCA1.