Former Regenxbio Team Launches Tern for Batten Disease Gene Therapies

Tern Therapeutics launched on August 27, securing $15 million in funding to support its development of two early-stage gene therapies targeting different symptoms of ceroid lipofuscinosis type 2 (CLN2), a rare neurodegenerative disease. This new biotech company’s candidates, along with its leadership team, originate from Regenxbio.

Tern Therapeutics holds global licenses for TTX-381 and TTX-181, previously known as RGX-381 and RGX-181, respectively. Their lead program, TTX-381, is an adeno-associated viral (AAV) vector-based gene therapy aimed at addressing vision loss in CLN2 patients by delivering a copy of the TPP1 gene directly to the retina. A Phase I/II dose-escalation study is currently being conducted in London and plans to enroll up to 16 children aged between 1 and 12 years. The study is projected to conclude by mid to late 2025.

The second asset, TTX-181, employs an AAV vector to deliver the gene therapy directly to the central nervous system (CNS) to prevent further neurodegeneration associated with the disease. Two years ago, a single child received this experimental treatment at the Hospital de Clinicas in Porto Alegre, Brazil, as part of an investigator-initiated study. As of December 2022, this therapy was well-tolerated, and no serious adverse events related to the treatment were reported.

The $15 million funding round was led by ATW Partners and Steve Oliveira, head of Nemean Asset Management. This financial support is expected to propel TTX-381 through its current clinical trial and further the development of TTX-181.

Tern Therapeutics not only derives its pipeline from Regenxbio but also its leadership. Alex Bailey, Tern’s Chief Executive Officer, previously headed early program and portfolio development at Regenxbio. Bailey expressed enthusiasm about continuing the work initiated at Regenxbio and emphasized the team’s deep familiarity with the programs. He highlighted the trust and confidence placed in them by Regenxbio and the patient community to lead the subsequent developmental phase of these promising therapies.

Bailey co-founded Tern Therapeutics alongside Christina Ohnsman and Matthew Rosini. Ohnsman serves as Tern’s Chief Medical Officer and was formerly the executive director of clinical development at Regenxbio. She managed the firm’s portfolio of gene therapies for ocular and CNS rare diseases. Before her tenure at Regenxbio, she was a medical consultant, providing commercialization advice for Luxturna (voretigene neparvovec-rzyl), the first FDA-approved gene therapy.

Matthew Rosini, Tern’s Chief Financial and Administrative Officer, was previously the head of strategic initiatives at Regenxbio. He also has experience as a partner at FoxKiser, a law firm focused on life sciences.

Amy Fenton Parker, president and CEO of the Batten Disease Support, Research, & Advocacy organization, expressed optimism about Tern’s launch. She underscored the importance of transitioning both gene therapy programs to a team already deeply familiar with the projects and connected to the CLN2 community. Parker believes this seamless transition is crucial for the continued advancement and clinical research of these therapies.

Through this strategic launch and substantial funding, Tern Therapeutics is positioned to make significant strides in the treatment of CLN2, offering hope to patients and families affected by this rare and debilitating condition.

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