Tern Therapeutics
Tern Therapeutics is dedicated to developing transformative, one-time gene therapy treatments for rare diseases, with a focus on conditions like CLN2 Batten disease. The company emphasizes urgency, innovation, integrity, empathy, and determination in its mission to improve the lives of patients worldwide.
Industries
Nr. of Employees
small (1-50)
Tern Therapeutics
Products
Investigational retina-directed gene therapy for CLN2-associated ocular disease
An investigational one-time gene replacement therapy designed to deliver a functional copy of the deficient gene to the retina to produce ongoing enzyme and potentially prevent or slow vision loss associated with CLN2 disease.
Investigational CNS-directed gene therapy for CLN2 neurological disease
An investigational gene replacement therapy designed for delivery to the brain via cerebrospinal fluid to produce ongoing enzyme within the CNS and address neurological degeneration in CLN2 disease.
Investigational retina-directed gene therapy for CLN2-associated ocular disease
An investigational one-time gene replacement therapy designed to deliver a functional copy of the deficient gene to the retina to produce ongoing enzyme and potentially prevent or slow vision loss associated with CLN2 disease.
Investigational CNS-directed gene therapy for CLN2 neurological disease
An investigational gene replacement therapy designed for delivery to the brain via cerebrospinal fluid to produce ongoing enzyme within the CNS and address neurological degeneration in CLN2 disease.
Services
Operation and oversight of clinical trials for investigational gene therapies, including site selection, enrollment logistics, and safety monitoring.
Provision of patient-facing communications channels, resources, and contacts to support information requests and community outreach.
Operation and oversight of clinical trials for investigational gene therapies, including site selection, enrollment logistics, and safety monitoring.
Provision of patient-facing communications channels, resources, and contacts to support information requests and community outreach.
Expertise Areas
- Ocular gene therapy development
- CNS-directed gene therapy
- Clinical trial management for rare diseases
- Regulatory strategy for advanced therapies
Key Technologies
- Gene replacement therapy
- Retina-targeted gene delivery
- CSF / intracerebroventricular gene delivery
- Clinical trial design and registration