Atsena Therapeutics Secures $150M Series C Funding for Ocular Gene Therapies

Atsena Therapeutics, a North Carolina-based biotech company specializing in gene therapies for inherited retinal diseases, has successfully raised $150 million in an oversubscribed Series C financing round. The funding, led by new investor Bain Capital, will primarily support the development of the company's lead program, ATSN-201, a potential treatment for X-linked retinoschisis (XLRS).

Funding Details and Investor Lineup

The impressive $150 million raise marks a significant step up from Atsena's previous funding rounds, which included a $55 million Series A in 2020 and a $24 million Series B. The current round saw participation from a roster of notable investors, including Sofinnova Investments, Abingworth, Wellington Management, Lightstone Ventures, Foundation Fighting Blindness, Hatteras Venture Partners, Osage University Partners, and the Manning Family Foundation.

ATSN-201: Targeting X-linked Retinoschisis

ATSN-201, Atsena's lead candidate, is designed to treat or prevent blindness caused by XLRS, an inherited eye condition primarily affecting males and typically diagnosed in childhood. The gene therapy has already received fast track, rare pediatric disease, and orphan drug designations from the FDA.

Currently, ATSN-201 is being evaluated in the Lighthouse phase 1/2 trial. The study has progressed to its second portion, with nine adults having been treated at three dose levels in the initial part. No serious adverse events were reported in the first phase. The trial continues to recruit patients over six years old for a one-time subretinal injection of ATSN-201 in one eye, with plans to include both pediatric and adult participants in the second part.

Expanding the Pipeline and Technology Platform

The new funding will also support Atsena's preclinical pipeline and the expansion of its proprietary spreading AAV.SPR capsid technology. This innovative approach, utilized in ATSN-201, is designed to target previously unreachable areas of the retina while avoiding the surgical risks associated with foveal detachment.

Additionally, Atsena is advancing ATSN-101, a clinical-stage gene therapy for Leber congenital amaurosis type 1. The therapy has completed a phase 1/2 trial with promising safety results and has received rare pediatric disease, orphan drug, and regenerative medicine advanced therapy designations from the FDA. Atsena is now collaborating with Japanese pharmaceutical company Nippon Shinyaku to progress ATSN-101 towards a global pivotal study.

Industry Context and Challenges

The substantial funding round for Atsena comes at a challenging time for the cell and gene therapy sector. The first quarter of 2025 saw significant layoffs in the industry, with cell and gene therapy companies accounting for approximately 31% of biopharma job cuts. Recent events, such as Roche's restructuring of its Spark gene therapy unit and Pfizer's withdrawal of its FDA-approved hemophilia B gene therapy Beqvez from the market, underscore the evolving landscape and challenges faced by gene therapy developers.

Despite these industry headwinds, Atsena's successful fundraising demonstrates continued investor confidence in the potential of innovative gene therapies for ocular diseases. As the company advances its lead candidates through clinical trials, the outcomes of these studies will be closely watched by both the scientific community and industry observers.