Tern Therapeutics debuted on Tuesday with $15 million in funding and a pair of early-stage gene therapies designed to treat separate symptoms of the ceroid lipofuscinosis type 2 (CLN2) form of Batten disease, a rare neurodegenerative condition. Both candidates, as well as the biotech’s leadership team, can trace their roots to Regenxbio. The newco has global licences to TTX-381 and TTX-181 — formerly RGX-381 and RGX-181, respectively — from Regenxbio. The most advanced programme, TTX-381, is an adeno-associated viral (AAV) vector-based gene therapy engineered to deliver a copy of the TPP1 gene directly to the retina, with the aim of treating the vision loss associated with CLN2 disease. A Phase I/II dose-escalation study is underway in London. According to ClinicalTrials.gov, the trial will enrol up to 16 children between the ages of 1 and 12 and is expected to wrap in mid- to late 2025. The second asset, TTX-181, is meant to be delivered via AAV vector directly to the central nervous system (CNS) to prevent further neurodegeneration caused by the disease. Two years ago a child was given the experimental treatment at the Hospital de Clinicas in Porto Alegre, Brazil as part of a single-patient, investigator-initiated study. The gene therapy was well-tolerated, with no treatment-related serious adverse events as of December 2022.The financing, which was led by ATW Partners and Steve Oliveira, head of Nemean Asset Management, is expected to help move TTX-381 through its current trial and advance TTX-181. Gene therapy experienceNot only does Tern’s pipeline hail from Regenxbio, but so do its founders. Chief executive Alex Bailey previously was the head of early program and portfolio development at the Maryland-based firm.The Tern team is “excited to continue the work we started at Regenxbio,” he commented. “No one knows these programmes 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,” Bailey added.He founded the biotech along with Christina Ohnsman and Matthew Rosini, who serve as Tern’s chief medical officer and chief financial and administrative officer, respectively. Ohnsman was executive director of clinical development at Regenxbio, where she oversaw the firm’s portfolio of ocular and CNS rare disease gene therapies. Prior to joining the drug developer, she was a medical consultant and provided advice on commercialisation initiatives for Luxturna (voretigene neparvovec-rzyl), the first-ever FDA-approved gene therapy. At Regenxbio, Rosini served as head of strategic initiatives; he previously was a partner at the life sciences-focussed FoxKiser law firm.Amy Fenton Parker, president and CEO of the Batten Disease Support, Research, & Advocacy organisation, called the launch of Tern “incredibly encouraging for our CLN2 community.”“The transition of both gene therapy programs to the Tern Therapeutics team, who are already so familiar with the programs and connected to our CLN2 community, is invaluable for the seamless continuity and advancement of this clinical research,” she said.
