Tern Therapeutics Launches with $15 Million and Pipeline in CLN2 Batten Disease

Rare Daily Staff

Tern Therapeutics launched with a $15 million financing and a global licensing agreement with RegenxBio for two experimental gene therapies to treat manifestations of CLN2 disease, a form of the ultra-rare and deadly neurodegenerative disorder Batten Disease.

ATW Partners and biotech investor Steve Oliveira, head of Nemean Asset Management, led the investment.

The deal follows cuts at RegenexBio late last year as the company sought partners for its programs in neurodegeneration. The deal with Tern provides a path forward for two of its experimental gene therapies.

Tern co-founder and CEO Alex Bailey previously served as head of early program and portfolio development for RegenexBio and prior to that he had a leadership roled at the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research. He is joined by co-founders and RegenxBio alums including Tern Chief Medical Officer Christina Ohnsman, who served as executive director of clinical development at RegenxBio and Chief Financial and Administrative Officer Matthew Rosini, who was head of strategic initiatives at RegenxBio.

“We’re driven to deliver transformative treatments to patients living with rare diseases and are excited to continue the work we started at REGENXBIO,” said Bailey. “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 to lead the next phase of development of these promising investigational therapies.”

Tern said patient communities are central to Tern’s approach to the development of investigational therapies, including partnerships with the Batten Disease Family Association; Batten Disease Support, Research, & Advocacy; BDSRA Australia; and other patient advocacy organizations.

Tern licensed TTX-381, an experimental one-time AAV gene therapy designed to deliver a working copy of the TPP1 gene directly to the retina, potentially providing a durable source of TPP1 activity intended to maintain the health of the retina and address vision loss in people with CLN2 disease. The safety and tolerability of TTX-381, as well as its effect on retinal anatomic and functional outcomes, are being evaluated in a first-in-human, open-label, dose escalation phase 1/2 clinical trial at Great Ormond Street Hospital in London, United Kingdom.

The other gene therapy it licensed is TTX-181, an experimental, one-time AAV gene therapy designed to deliver a working copy of the TPP1 gene directly to the central nervous system, potentially providing sustained levels of TPP1 intended to prevent worsening of neurological degeneration in people with CLN2 disease. In late 2022, physician investigators at the Hospital de Clinicas in Porto Alegre, Brazil, dosed a child with CLN2 Batten disease with TTX-181 in a single-patient, investigator-initiated study.