Positive early safety, efficacy data reported for Leber congenital amaurosis gene therapy

Leber congenital amaurosis (LCA) is a family of congenital retinal dystrophies that results in severe vision loss at an early age. LCA can be caused by mutations in at least 29 genes, with mutations in GUCY2D responsible for about 12% of cases. 

Atsena Therapeutics reported positive 12-month safety and efficacy data from a phase 1/2 trial of ATSN-101 gene therapy in GUCY2D-associated LCA. During the trial, fifteen patients received injections of ATSN-101 under the retina of one eye. 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.

No serious treatment-emergent adverse events were seen at 12 months, according to a press release. In addition, patients who received a high dose of ATSN-101 experienced an improvement in best corrected visual acuity. Some patients had 10,000-fold improvement in retinal sensitivity.

“The durability of clinically meaningful visual improvements in the absence of serious treatment-related adverse events at the 12-month mark underscore the safety, tolerability and efficacy of our subretinal gene therapy,” Kenji Fujita, MD, chief medical officer of Atsena, said in the release. “We believe the 12-month findings provide solid proof of concept that ATSN-101 will exceed the requirements set by the U.S. Food and Drug Administration for ultimate approval. We are exploring partnering and out-licensing options to advance ATSN-101 into a pivotal trial.”

For more information about this trial, visit https://clinicaltrials.gov/study/NCT03920007. This trial is active but no longer recruiting.

Source: Healio Ocular Surgery News, December 6, 2023; see source article