Tern Therapeutics Opens New TTX-381 Clinical Trial Site at UKE in Hamburg, Germany
First clinical trial site for Tern Therapeutics in the European Union
Approval of CTA from Germany’s federal health authority PEI
WASHINGTON, DC, October 28, 2025 — Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, today announced the approval of a clinical trial application (CTA) from Germany’s federal health authority, Paul-Ehrlich-Institut (PEI), and the opening of a second clinical trial site to evaluate the safety and efficacy of TTX-381, its novel one-time gene therapy candidate for the treatment of the ocular manifestations of CLN2 Batten disease. The newly initiated site is University Medical Center Hamburg Eppendorf (UKE) in Hamburg, Germany, a leading global center for the study and treatment of Batten disease.
UKE is the second clinical trial site for the ongoing TTX-381 study, joining Great Ormond Street Hospital (GOSH) in the United Kingdom. Both UKE and GOSH have world-renowned experts that provide specialized and comprehensive care for patients with CLN2 disease and have conducted pioneering research in the development of new therapies. As the center with the largest cohort of CLN2 patients worldwide, UKE has conducted many studies of the natural history, evaluation, and response to treatment of this rare, progressive neurodegenerative disorder. "The UKE team have been long-time collaborators on the TTX-381 and TTX-181 programs. We are thrilled to welcome them as a TTX-381 clinical trial site and look forward to deepening our partnership with them in bringing this desperately needed treatment to children living with CLN2," said Christina Ohnsman, MD, Chief Medical Officer of Tern. “We are also profoundly grateful to the GOSH team for their ongoing commitment to enrolling this trial with unprecedented speed.”
"We are excited to become a study center for TTX-381, an innovative gene therapy designed to prevent blindness in children with CLN2 disease. This one-time treatment delivers a functional TPP1 gene directly to the retina, offering hope to young patients facing progressive vision loss. As a leading European center for CLN2 research, we are proud to contribute to this groundbreaking trial and bring potential new treatment options to the children and families we serve,” said Dr. Angela Schulz, MD, PhD, Head of the Research Group for Childhood Neurodegenerative Disease of UKE and Principal Investigator for the UKE site.
The open-label clinical trial designed to evaluate the safety and efficacy of TTX-381 is active and recruiting participants. Learn more at euclinicaltrials.eu (EUCT number: 2025-52117531-00) and clinicaltrials.gov (NCT05791864).
About TTX-381
TTX-381 is an investigational one-time AAV gene therapy designed to deliver a working copy of the TPP1 gene directly to the retina to provide a durable source of TPP1 to maintain the health of the retina and address vision loss in people with CLN2 disease. Vision loss in CLN2 disease rapidly progresses to blindness, and there is currently no available treatment for the ocular manifestations of CLN2 disease.
About Tern Therapeutics
TERN THERAPEUTICS is a privately held biotechnology company founded in 2023 with a new vision for speeding the development of transformative, one-time gene therapy medicines for rare diseases. Guided by a team of leading physicians, scientists, and business leaders and in collaboration with patient communities, Tern is driven to deliver these transformative treatments with urgency to those living around the world with rare diseases. For more information about Tern, please visit www.terntx.com.
Contacts
Matthew Rosini
Tern Therapeutics, LLC
mrosini@terntx.com
