Two Regenxbio gene therapies are getting a second chance at newly-seeded startup Tern Therapeutics, which launched Tuesday with $15 million.
In November, Regenxbio announced it was laying off 15% of 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.
The two Batten disease gene therapies 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 2023 and is led by 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.)
“No one knows these programs better than the team we’ve assembled at Tern, and we deeply appreciate the confidence and trust that Regenxbio and the patient community have placed in us,” Bailey said in a press statement.
The $15 million investment, led by ATW Partners and head of Nemean Asset Management 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 second AAV gene therapy licensed by Tern delivers a functional copy of the TTP1 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.