New Hope on the Horizon: Tern Therapeutics Launches with Focus on Rare Disease Gene Therapies
The inherited retinal disease (IRD) community is always looking for new advancements and dedicated efforts in gene therapy. A new biotechnology company, Tern Therapeutics, has recently launched, bringing with it a renewed focus on developing transformative, one-time gene therapies for rare diseases, including those with ocular manifestations. This launch is particularly noteworthy as it is led by former executives from Regenxbio, a company known for its work in gene therapy.
Tern Therapeutics officially launched on August 27, 2024, securing $15 million in financing. This funding will be used to accelerate ongoing clinical testing and advance its therapeutic pipeline. The company was founded in late 2023 by Alex M. Bailey, PhD, who serves as CEO, Christina Ohnsman, MD, Chief Medical Officer, and Matthew Rosini, Chief Financial and Administrative Officer. All three held leadership positions at Regenxbio, with Dr. Bailey previously serving as Head of Early Program and Portfolio Development, Dr. Ohnsman as Executive Director of Clinical Development, and Mr. Rosini as Head of Strategic Initiatives.
A key aspect of Tern Therapeutics' launch is a global licensing agreement with Regenxbio for two experimental gene therapies, now designated TTX-381 and TTX-181. These therapies are specifically aimed at treating manifestations of CLN2 Batten disease, a rare and severe neurodegenerative disorder. While CLN2 Batten disease primarily affects neurological function, it also includes significant ocular manifestations, with vision loss being a common symptom that can progress to blindness.
One of the lead candidates, TTX-381, is an investigational one-time AAV gene therapy designed to deliver a working copy of the TPP1 gene directly to the retina. The goal of TTX-381 is to provide a durable source of the TPP1 enzyme to maintain retinal health and address vision loss in individuals with CLN2 disease. This therapy is currently being evaluated in a first-in-human, open-label, dose escalation Phase 1/2 clinical trial at Great Ormond Street Hospital in London, United Kingdom. The second gene therapy, TTX-181, is designed to deliver the TPP1 gene to the brain to prevent the progression of neurological degeneration.
For patients and families affected by IRDs, the launch of Tern Therapeutics signifies continued dedication and investment in gene therapy research for rare eye conditions. The team's commitment to advancing these programs, even after their previous company deprioritized them, underscores a deep personal commitment to the patient community. This focus on ocular manifestations within a broader rare disease context highlights the interconnectedness of various rare conditions and the potential for gene therapy to offer hope where no treatments currently exist for certain aspects of these diseases.
Tern Therapeutics emphasizes that patient communities are central to their approach, and they have already established partnerships with several Batten disease patient advocacy organizations. This patient-centric model is crucial for ensuring that new therapies are developed with the needs and perspectives of those living with the disease at the forefront. The company aims to deliver transformative treatments with urgency to those living with rare diseases.
The ongoing clinical trial for TTX-381 represents a tangible step forward. The continued development of gene therapies like TTX-381 offers a beacon of hope for addressing the severe vision loss associated with conditions like CLN2 Batten disease, and potentially informing future research in other IRDs.