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.
https://globalgenes.org/raredaily/tern-therapeutics-launches-with-15-million-and-pipeline-in-cln2-batten-disease/
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