There’s still money to be raised by gene therapy startups with good clinical data.

On Wednesday, North Carolina biotech Atsena Therapeutics said it had raised a $150 million Series C to try and be the first to treat a rare form of ocular disease that leads to blindness.

Bain Capital Life Sciences led the financing. Another new Atsena investor, Wellington Management, also took part as did all existing backers, including Lightstone Ventures, Sofinnova Investments and Abingworth, among others.

“There is a place here for programs and companies that do have compelling clinical data in diseases with high unmet clinical needs,” Atsena CEO Patrick Ritschel said in an interview.

The gene therapy field has been held back by struggles with commercialization, pipeline cuts and funding pullbacks. Only a few ocular gene therapy companies have been able to secure nine-figure financings since 2022, including Beacon, Ray and Frontera. Atsena last disclosed a $24.5 million Series B in October 2023.

Funding through a BLA

Atsena is working on Part B of a Phase 1/2 study of ATSN-201, a gene therapy that it hopes will treat a genetic ocular disease called X-linked retinoschisis, or XLRS. The condition is usually found in childhood and impacts about 30,000 men in the US and European Union, the biotech said.

“In a majority of the nine patients treated, we see anatomic resolution of disease and we see functional improvement in the majority. So it’s not often you can get that kind of convincing signal on a small dataset,” Leonard Feiner, a principal at Bain Capital Life Sciences, said in an interview.

The disease causes an abnormal split in the retinal layers and can’t be fixed by glasses, eventually leading to blindness.

“The interesting thing about this disease, XLRS, is that in addition to having functionally decreased vision, these patients also have an anatomic problem, which is a lack of adhesion between the layers of the retina,” Feiner said.

The therapy is a subretinal injection and uses an adeno-associated virus.

The new funding is slated to carry Atsena all the way through a biologics license application at the FDA “and beyond,” Ritschel said. The startup will meet with the FDA later this year to clarify if the Phase 1/2 could support an accelerated approval, or if the biotech would need to run an additional study. The future of cell and gene therapy decisions at the FDA is now in limbo as top leadership, most notably Peter Marks, has been forced out or resigned in recent days.

“We’ve seen others in this space in [the past] 12 months or so obtain accelerated approval alignment with the FDA in rare disease, genetic therapy environments, so we would be hopeful for that as well,” Ritschel said.

It’s not the first company to try and treat the disease. Biogen had a $1 billion-plus pact with Applied Genetic Technologies Corporation, but their gene therapy failed a Phase 1/2 study in 2018.

Atsena is also working on ATSN-101 for Leber congenital amaurosis type 1, or LCA1. The biotech began searching for a partner for the former Sanofi gene therapy in 2023 and shortly after presented one-year data from a Phase 1/2 trial. It secured a licensing agreement with Kyoto-based Nippon Shinyaku last fall.

Ritschel said the pivotal study has yet to begin and declined to disclose any expected timing.

The Series C will also support a preclinical pipeline of gene therapies for inherited retinal diseases like MYO7A-associated Usher syndrome, or USH1B, the company said. Atsena employs “just over 20 people today,” Ritschel said.

Prior to joining the company in 2020, he was involved in other gene therapy upstarts as co-founder of StrideBio and an angel investor in Bamboo Therapeutics. Ginkgo bought AAV capsid assets from StrideBio in 2023 and Pfizer acquired Bamboo for up to $645 million in 2016.

AUTHOR

Kyle LaHucik

Senior Reporter

[email protected]