VIDEO: Leber congenital amaurosis gene therapy promising in phase 1/2 trial
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Key takeaways:
- High-dose ATSN-101 showed early positive results for the treatment of Leber congenital amaurosis.
- No drug-related ocular serious adverse events were reported.
NEW ORLEANS — In this Healio Video Perspective from the ARVO meeting, Christine Kay, MD, discusses 6-month results investigating ATSN-101 for the treatment of Leber congenital amaurosis caused by mutations in the GUCY2D gene.
“In the ongoing phase 1/2 gene therapy trial, there were positive clinical results in patients treated at the highest dose with ATSN-101,” she said.
The nine patients who received the highest dose experienced clinically significant improvements in full-field stimulus testing from baseline compared with untreated eyes, according to Kay.
There were no drug-related ocular severe adverse events reported in the trial.
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Kay C, et al. Six-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). Presented at: Association for Research in Vision and Ophthalmology meeting; April 23-27, 2023; New Orleans.
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