Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, reported positive twelve-month clinical data relating to the safety and efficacy of its TTX-381 program at the 19th International Congress on Neuronal Ceroid Lipofuscinosis (NCL) in Queensland, Australia.

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.

Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, today announced that TTX-381 has been awarded the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) innovative medicine designation. TTX-381 is a novel one-time investigational gene therapy product being developed for the treatment of the ocular manifestations of CLN2 disease, a form of Batten disease.

Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to TTX-381. TTX-381 is a novel one-time gene therapy product being developed for the treatment of the ocular manifestations of CLN2 disease, a form of Batten disease.

Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to TTX-381. TTX-381 is a novel one-time gene therapy product being developed for the treatment of the ocular manifestations of CLN2 disease, a form of Batten disease.

Dear CLN2 Batten Community, We are pleased to share updates on Tern Therapeutics' progress with our clinical trials for TTX-381 and TTX-181, which are investigational gene therapies being developed for the treatment of CLN2 Batten disease. These therapies aim to address both the vision loss and neurological symptoms.

Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, reported the achievement of key development milestones for its programs TTX-381 and TTX-181, as well as presented positive clinical data relating to the safety and efficacy of both programs on February 7, 2025 at the 21st Annual WORLDSymposium™ in San Diego, California. TTX-381 and TTX-181 are novel one-time gene therapy products being developed for the treatment of the ocular and central nervous system (CNS) manifestations of CLN2 disease, a form of Batten disease.

Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, today announced its launch and closing of a $15 million financing. Concurrently, Tern also announced that it has entered into a global licensing agreement with REGENXBIO Inc. ("REGENXBIO") for RGX-381 and RGX-181 (now designated TTX-381 and 181, respectively) to form its initial therapeutic pipeline.