A Durham-based biotechnology firm, Atsena Therapeutics, is making strides in developing innovative treatments for a rare form of blindness, having recently raised $150 million in funding. This financing round, led by Bain Capital, aims to advance Atsena's lead gene therapy program, ATSN-201, which targets X-linked retinoschisis, a hereditary retinal disease affecting approximately 30,000 individuals in North America and Europe.

X-linked retinoschisis typically manifests in childhood and can lead to significant vision loss as individuals age. The disease causes separations between the layers of the retina, resulting in debilitating visual impairment. Atsena's CEO, Patrick Ritschel, emphasized the urgent need for effective treatments, as current options are virtually nonexistent.

The company has reported promising early-stage clinical results for ATSN-201, demonstrating its ability to close the schisis cavities—gaps in the retinal layers. In the initial phase of a Phase 1/2 clinical study involving nine patients, the therapy not only showed structural improvements but also enhancements in visual function, even in areas beyond the treatment site.

These positive outcomes have attracted significant investor interest, including Bain Capital. Dr. Leonard Feiner, a principal on Bain's life sciences team, noted the strong efficacy signals and safety data from the small patient cohort. He highlighted the unmet medical need that Atsena is addressing, describing the gene therapy approach as particularly suitable for this condition.

The Series C funding round also saw participation from new investor Willington Management, alongside existing backers such as Lightstone Ventures, Sofinnova Investments, and the Foundation Fighting Blindness. This financial boost will enable Atsena to continue its clinical studies, including a second phase involving 12 additional patients, and to prepare for a pivotal trial and potential commercialization upon regulatory approval.

In addition to the funding for ATSN-201, Atsena recently entered an exclusive licensing agreement with Nippon Shinyaku Co. for another gene therapy program targeting Leber congenital amaurosis 1 (LCA1). This agreement grants Nippon Shinyaku commercial rights in the U.S. and Japan while allowing Atsena to retain rights in other regions. The deal includes an upfront payment and potential milestone payments and royalties based on future sales.

Overall, Atsena Therapeutics is positioned to make significant advancements in the treatment of rare retinal diseases, with its innovative gene therapy programs showing promise in early clinical trials. The recent funding will support the company's ongoing research and development efforts, aiming to bring effective therapies to patients in need.