Atsena’s LCA1 Gene Therapy Licensed by Nippon Shinyaku for Markets in US and Japan

Nippon Shinyaku, a pharmaceutical company based in Kyoto, Japan, and Atsena Therapeutics, a developer of gene therapies for retinal diseases in Durham, North Carolina, have entered into a license agreement for the commercialization of ATSN-101, an emerging gene therapy for Leber congenital amaurosis 1 (LCA1) which is caused by mutations in the gene GUCY2D. Under the terms of the agreement, Nippon Shinyaku will receive exclusive rights to commercialize ATSN-101 in the US and Japan, and Atsena will retain commercial rights for the rest of the world. A Phase 3 global clinical trial for ATSN-101 is planned.

The RD Fund, the venture philanthropy arm of the Foundation Fighting Blindness, is a major investor in Atsena. It also helped form the company and played a pivotal role in Atsena’s licensing of the LCA1 program from Sanofi. 

“We are delighted with the Atsena and Nippon Shinyaku partnership to move ATSN-101 into a pivotal, Phase 3 clinical trial. It’s especially meaningful to the field and the affected community that we advance this gene therapy program toward regulatory approval,” says Rusty Kelley, PhD, managing director of the RD Fund. “Excitingly, results from the Phase 1/2 clinical trial show that the gene therapy has real promise for restoring vision to people with severe vision loss from LCA1.”

In October 2022, Atsena announced positive results from its 15-participant, Phase 1/2 clinical trial for ATSN-101. Overall, the gene therapy 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. Those patients could also better navigate a multi-luminance mobility course. One teenager was able to see snowflakes for the first time after receiving ATSN-101.

ATSN-101 is administered through the injection of a tiny drop of liquid underneath the retina. The gene therapy uses an engineered virus, which works like a shipping container system, to deliver healthy copies of the GUCY2D gene 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, PhD, and Founder and Chief Technology Officer Sanford Boye, MSc, at the University of Florida.

ATSN-101 has received a Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food & Drug Administration. The designation provides the opportunity for priority review and accelerated approval for the treatment.