Two Regenxbio gene therapies are getting a second chance at newly-seeded startup Tern Therapeutics, which launched from stealth Tuesday with $15 million.
In November, Regenxbio announced it was laying off 15% of its staff and looking for others to take on its neurodegenerative gene therapy programs, including a treatment for mucopolysaccharidosis type I — a disease in which people can’t make enough of a key enzyme to break down sugars — and two therapies for Batten disease.
Tern is taking on the two Batten disease gene therapies, which are for the late-infantile form of the disease known as CLN2. In Batten disease, children’s nervous system function progressively declines, leading to seizures and vision loss.
Alex BaileyTern was founded in late 2023 and is currently a small startup of three — Alex Bailey, Christina Ohnsman and Matthew Rosini — who all formerly held leadership positions at Regenxbio. Bailey was head of early program and portfolio development and now serves as CEO at Tern. (A separate, publicly-traded company called Terns Pharmaceuticals develops small molecule drugs.)
Bailey told Endpoints News that after Regenxbio’s decision to deprioritize these gene therapy programs, “within weeks by early December” they began discussing with Regenxbio and patient groups how to move the experimental therapies forward. “We designed these programs, we built these teams, and we were hitting every metric we possibly could want, in terms of early efficacy and bioactivity,” Bailey said. “We were at this key moment where the programs were deprioritized for strategic reasons and not due to underlying science.”
“As we started to generate data and saw how promising they were, we couldn’t let the product expire in a freezer,” said Ohnsman, who is now Tern’s CMO. “We felt like we had this commitment to the patient community, and we wanted to see it through.”
The $15 million investment, led by ATW Partners and Nemean Asset Management head Steve Oliveira, will fund an ongoing clinical study of an AAV gene therapy that’s delivered to the retina for vision loss associated with the CLN2 Batten disease. The therapy, dubbed TTX-381, is currently being tested in an early-stage study at the Great Ormond Street Hospital in London. The goal, Bailey said, is to get this therapy through to a marketing application with their funding.
The second AAV gene therapy licensed by Tern delivers a functional copy of the TPP1 gene — the gene that’s mutated in CLN2 Batten disease — to the central nervous system in hopes of preventing the progression of neurological degeneration. The therapy was tested in 2022 in a single child with the disease as part of an investigator-initiated study in Brazil.
“For a while, rare diseases were kind of the sexy target, and then when the economy shrank, that became much more challenging. We see that across the industry, with many of these programs being discontinued or just paused,” Ohnsman said. “It’s difficult to justify, when you have limited resources in any company, to put your resources behind something that isn’t going to have a big return on your investment, but it’s everything to the communities who are suffering with these rare diseases.”