Atsena Therapeutics and Nippon Shinyaku have announced an exclusive license agreement to advance ATSN-101, an investigational gene therapy for Leber congenital amaurosis (LCA) caused by biallelic mutations in the GUCY2D gene (LCA1). The collaboration aims to bring a new therapeutic option to patients suffering from this inherited retinal disease.
Under the agreement, Nippon Shinyaku gains exclusive commercial rights for ATSN-101 in the United States and Japan. Atsena Therapeutics will retain commercial rights in all other regions globally. In the U.S., NS Pharma Inc., a subsidiary of Nippon Shinyaku, will be responsible for marketing the gene therapy.
Atsena Therapeutics is set to receive an upfront payment, milestone payments, and royalties on sales. Additionally, the company will be reimbursed for ongoing development efforts, including a planned global pivotal trial of ATSN-101. This financial support will help accelerate the development and potential approval of the therapy.
ATSN-101: A Novel Gene Therapy for LCA1
ATSN-101 is a first-in-class gene therapy designed to treat LCA1, a genetic form of blindness. The therapy has received several designations from the U.S. Food and Drug Administration (FDA), including Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy (RMAT) Designation, and Orphan Drug Designation. These designations highlight the unmet need and potential of ATSN-101 to address this rare condition.
If Atsena Therapeutics receives a Rare Pediatric Disease Priority Review Voucher (PRV) following the approval of ATSN-101’s Biologics License Application (BLA), the company will retain all rights to the PRV, further incentivizing the development of this important therapy.
Leadership Perspectives
Patrick Ritschel, CEO of Atsena Therapeutics, emphasized that the agreement creates a path to accelerate the development of ATSN-101 and validates Atsena’s pioneering technology. He anticipates this collaboration will lead to more ocular gene therapy treatments from their clinical portfolio.
Nippon Shinyaku President Toru Nakai highlighted that ATSN-101 offers a potential innovative treatment in an area where no approved solutions currently exist. He expressed excitement about the collaboration with Atsena and its groundbreaking science.
