Atsena prioritizes retinal gene therapy pipeline, trims staff as existing investors refuel the biotech

Atsena prioritizes retinal gene therapy pipeline, trims staff as existing investors refuel the biotech

Atsena Therapeutics has reduced its staff, is looking for a partner and has reeled in more cash from existing investors, the gene therapy biotech told Endpoints News.

Patrick Ritschel

The North Carolina startup anticipates 12-month results from its first Phase I/II clinical trial by year’s end and began a separate Phase I/II study of its second gene therapy in August, but has recently laid off employees to conserve cash, which includes a recent insider-led Series B, the company said, with about $24.5 million of $32 million raised so far, per an SEC filing. CEO Patrick Ritschel declined to disclose the size of the workforce reduction.

Like many other biotechs, gene therapy startups have struggled in the current market environment, with impacts on headcount and pipeline plans. In Atsena’s field of ophthalmology and retinal disease, there has also been an influx of new entrants like Ray, Beacon, Complement, Eudora and Avista, among others.

Atsena came to the fore in December 2020 with a $55 million Series A from Sofinnova Investments, Abingworth, Lightstone Ventures, Hatteras Venture Partners, Osage University Partners, Manning Family Foundation and the Foundation Fighting Blindness’ RD Fund. The biotech “is in ongoing discussions with potential additional investors,” Atsena said in a statement.

The biotech was founded out of the labs of University of Florida couple Shannon Boye and Sanford Boye, a pair of leaders in ocular gene therapy. Their work in the early 2000s led Sanofi to license the treatment candidates, and Atsena was then formed in 2020 to license them back for further clinical testing.

Atsena’s first candidate, ATSN-101, goes after GUCY2D-associated Leber congenital amaurosis, or LCA. The condition impacts about 20% of people with the LCA group of inherited retinal diseases, for which there are no approved treatments, the biotech has said. At the time of a six-month interim data readout in April, “clinically meaningful improvements in visions” were seen at the highest dose, the startup said.

In a statement, the CEO said “Atsena is exploring options to partner or out-license the program.”

Their next candidate, ATSN-201, entered human studies in August for treating X-linked retinoschisis (XLRS) and will be the priority going forward, the company said, with the new money expected to get the company to topline results of a Phase I/II study.

XLRS mainly affects boys and men, impacting about 30,000 in the US and EU, per the biotech. No treatments exist for the rare condition, which leads to retinal splitting, impaired sight and progressive vision loss. In preclinical studies, Atsena said its AAV spreading capsids did not lead to inflammation and helped the treatment go beyond the subretinal injection site.

Beyond those two gene therapies, Atsena is working on ATSN-301 for MYO7A-associated Usher syndrome, or USH1B. The inherited condition is found in about 20,000 children in the US and EU who are born deaf and lose their sight within 10 years of birth. To treat these children, Atsena said it needed to create a dual AAV vector-based gene therapy because of the genetic payload’s large size.

Three additional, undisclosed programs are listed on Atsena’s pipeline.

This content was originally published here.