Atsena’s LCA-GUCY2D Gene Therapy Improves Vision in Phase 1/2 Clinical Trial

Atsena Therapeutics, a gene therapy development company focused on preventing and reversing blindness, announced positive results from its Phase 1/2 gene therapy clinical trial for people with Leber congenital amaurosis type 1 (LCA1), which is caused by mutations in the gene GUCY2D. Data from the trial was presented on October 1, 2022, at the American Academy of Ophthalmology Annual Meeting in Chicago.

The company reported results for 15 trial participants. Overall, the gene therapy, ATSN-101, was well tolerated. The nine patients receiving the highest dose of ATSN-101 had clinically meaningful vision improvements as measured by a full-field stimulus test (FST), which measures the patient’s ability to respond to different levels of light, and by their ability to navigate a multi-luminance mobility course.

Kenji Fujita, MD, Atsena’s chief medical officer, said, “We’re encouraged by these data that demonstrate ATSN-101 improved visual function while maintaining a favorable safety profile. We look forward to launching a pivotal trial for the evaluation of ATSN-101, which will lay the groundwork for successful registration and commercialization.”

ATSN-101 is administered through the injection of a tiny drop of liquid underneath the retina. The gene therapy uses a human-engineered virus, which works like a vast shipping container system, to deliver healthy copies of GUCY2D to the patient’s retinal cells to augment the mutated copies.

The GUCY2D gene therapy was created in the laboratory of Atsena Founder and Chief Scientific Officer Shannon Boye, Ph.D., and Founder and Chief Technology Officer Sanford Boye, M.Sc., at the University of Florida.

Atsena is being funded by an investment from the Foundation’s RD Fund, a venture philanthropy fund for emerging treatments in, or moving toward, early-stage clinical trials.

"Atsena’s early clinical proof of concept for improving vision in pediatric and adult LCA1 patients is great news for our field," said Rusty Kelley, PhD, managing director of the RD Fund. "Improvements in mobility and retinal sensitivity bring hope to affected LCA1 families and other stakeholders. The Foundation, the RD Fund, and our co-investors are bringing to bear all of the necessary resources to potentially enable a clinical and financial return for underserved indications including LCA1."

LCA is the most common cause of blindness in children, impacting two to three per 100,000. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. LCA1-GUCY2D is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this inherited retinal disease. Despite significant vision loss, people with GUCY2D mutations often have remaining retinal structure, making them good gene therapy candidates.

Atsena is also developing gene therapies for X-linked retinoschisis and Usher syndrome type 1B.

Read the complete press release.