Atsena and Nippon Shinyaku Enter Strategic Collaboration for ATSN-101 Advancement

Atsena Therapeutics has entered into an exclusive licensing agreement with Nippon Shinyaku for the commercialization of investigational gene therapy ATSN-101 for Leber congenital amaurosis. 

Atsena's ATSN-101 is a subretinal AAV5 gene therapy undergoing evaluation by phase 1/2 clinical trial specifically for the disease form caused by biallelic mutations in GUCY2D (LCA1). Currently, there are no approved treatments for it.

Under the new agreement, Nippon Shinyaku, based in Kyoto, Japan, will receive exclusive commercial rights in the US and Japan, and Atsena will retain commercial rights in the rest of the world. ATSN-101 will be marketed by NS Pharma, a wholly owned subsidiary of Nippon Shinyaku in the US.

“This collaboration creates a path to accelerate the development of ATSN-101 and validates Atsena’s pioneering technology and development capabilities,” Patrick Ritschel, CEO of Atsena Therapeutics said in a recent press release. “We look forward to working with Nippon Shinyaku as we advance ATSN-101 into a pivotal trial and potential approval to provide an innovative solution to patients and families affected by LCA1 around the world.”

The FDA has granted Atsena the Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation, and Orphan Drug Designation for ATSN-101, which aims to accelerate approval of safe and effective treatments for diseases affecting fewer than 200,000 people in the US. A recently published study in The Lancet suggests ATSN-101 maintains durable, clinically significant vision improvement at 12 months post-treatment.

“ATSN-101 provides a potential, innovative treatment in an area where no approved solutions currently exist,” Nippon Shinyaku President Toru Nakai said. “We are excited by the opportunity of this novel ocular gene therapy and our collaboration with Atsena and its groundbreaking science.”