Atsena Therapeutics' $150 million fundraise is a light of hope for the gene therapy field, which has suffered enormously in recent months.

Atsena Therapeutics has raised $150 million in an oversubscribed series C financing, with hopes that the funds can carry the biotech’s gene therapy designed to reverse or prevent blindness through a potential market approval.

The funding round was led by new investor Bain Capital, with participation from Sofinnova Investments, Abingworth, Wellington Management, Lightstone Ventures, Foundation Fighting Blindness, Hatteras Venture Partners, Osage University Partners and the Manning Family Foundation, according to an April 2 release.

The new cash is designed to fuel Atsena’s lead program, an investigational gene therapy dubbed ATSN-201. The asset is designed to treat X-linked retinoschisis (XLRS), an inherited eye condition that is typically diagnosed in childhood and can cause blindness. The genetic disease occurs primarily in males.

Atsena’s gene therapy has received fast track, rare pediatric disease and orphan drug tags from the FDA. The candidate is currently being studied in a phase 1/2 trial called Lighthouse, with updated data expected later this year.

In January, ATSN-201 entered the second portion of the study, with nine adults already treated at three dose levels in the first part. No serious adverse events were reported in the initial portion.

Atsena has already run a phase 1/2 trial for the therapy, with no serious treatment-emergent adverse events reported among the 15 enrolled patients. The candidate has collected rare pediatric disease, orphan drug and regenerative medicine advanced therapy designations from the FDA.

Atsena is now working to advance ATSN-101 toward a global pivotal study with partner Nippon Shinyaku, a pharma based in Japan.  

The $150 million financing is a big step up from Atsena’s $55 million series A raised back in 2020 and later a $24 million series B. In 2023, the biotech laid off an undisclosed number of employees to push forward development for ATSN-201.

The newest fundraise comes amid a reckoning for cell and gene therapy companies. The field suffered enormously in the first quarter of this year, with 20 of 63 biopharma layoff rounds occurring at cell and gene companies, representing about 31% of industry layoffs.

Several recent events—such as Roche’s overhaul of its Spark gene therapy unit or Pfizer pulling FDA-approved hemophilia B gene therapy Beqvez off the market—underscore the shift in how the industry views gene therapies. 

The study is still recruiting patients older than six years to receive a one-time subretinal injection of ATSN-201 in one eye. The second part of the trial will include pediatric participants as well as adults.

Currently, no approved treatments for XLRS exist.

The $150 million will also go toward Atsena’s preclinical pipeline and expand the use of the biotech’s spreading AAV.SPR capsid technology. The science is used for ATSN-201 and is designed to help reach part of the retina that can’t be treated with normal capsids while sidestepping the surgical risks of foveal detachment.

The North Carolina-based biotech also touts ATSN-101, a clinical-stage gene therapy for the treatment of Leber congenital amaurosis type 1, a severe inherited retinal disease.

By Gabrielle Masson  Apr 2, 2025 7:30am