Tern Therapeutics Launches with Batten Disease Gene Therapy Programs

Aug 28, 2024

Tern Therapeutics, a new biotech startup, officially launched on August 27, 2024, with $15 million in funding to advance gene therapies targeting Batten disease, specifically the ceroid lipofuscinosis type 2 (CLN2) form. This neurodegenerative disorder leads to vision loss and seizures in children. Tern’s pipeline includes two key gene therapy candidates, TTX-381 and TTX-181, both of which were originally developed by Regenxbio.
tern

Inherited from Regenxbio

Tern Therapeutics, founded by former Regenxbio leaders Alex Bailey, Christina Ohnsman, and Matthew Rosini, is focused on continuing the work they began at their former company. TTX-381, the most advanced candidate, is an adeno-associated viral (AAV) vector-based gene therapy designed to deliver a copy of the TPP1 gene directly to the retina, aiming to treat vision loss in CLN2 patients. This therapy is currently being evaluated in a Phase I/II clinical trial in the UK, involving up to 16 children aged 1 to 12. The trial is expected to conclude in 2025.

The second candidate, TTX-181, is also an AAV vector-based therapy but is delivered directly to the central nervous system (CNS) to halt neurodegeneration. This therapy showed promise in a 2022 investigator-initiated study in Brazil, where it was well-tolerated in a child with no serious adverse events reported.

 

Funding and Future Outlook

The $15 million funding round, led by ATW Partners and Steve Oliveira of Nemean Asset Management, is intended to support the ongoing trial of TTX-381 and advance TTX-181 further into development. The leadership at Terns, experienced in gene therapy from their time at Regenxbio, is committed to advancing these therapies, which they believe are crucial for the patient community suffering from rare diseases like CLN2.

As Tern Therapeutics embarks on this journey, the biotech aims to bring these promising therapies to market, providing new hope for children affected by Batten disease.

Source:
https://globalgenes.org/raredaily/tern-therapeutics-launches-with-15-million-and-pipeline-in-cln2-batten-disease/
GMP mRNA
Check out our AAV CDMO service to expedite your gene therapy research
About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Related Services

AAV Packaging Services

We have developed a series of proprietary technologies that greatly improve AAV production outcomes including titer, purity, potency, and consistency.

READ MORE

Off-the-Shelf AAV Products

We offer a library of carefully designed and pre-stocked AAV vectors for a wide variety of experimental needs.

READ MORE