Therapeutic Pipeline
Transformative AAV gene therapies for rare disease
The TTX-381 clinical trial is currently underway.
Clinical testing is being conducted at multiple sites around the globe for the treatment of ocular manifestations of CLN2 disease, a form of Batten disease.
CLN2 disease (neuronal ceroid lipofuscinosis type 2), a form of Batten disease, is a rare, pediatric-onset, genetic disorder caused by an inherited mutation, or error, in the CLN2 gene. The CLN2 gene contains instructions to make an enzyme called tripeptidyl peptidase 1, or TPP1. TPP1 helps to break down and remove waste material from cells in the body. Deficiency in TPP1 enzyme activity causes waste materials to build up in the body’s cells, particularly in the brain and the eye, where they cause progressive damage and can lead to seizures, rapid loss of language and motor function, cognitive decline, vision loss/blindness, and premature death.
What is CLN2 Disease?
How is TTX-381 Designed to Work?
Vision loss in CLN2 disease rapidly progresses to blindness, and there is currently no available treatment for the ocular manifestations of CLN2 disease. Tern Therapeutics is developing an investigational gene therapy, TTX-381, designed to deliver a working copy of the TPP1 gene to the retina so that it can make an ongoing supply of TPP1 enzyme to maintain the health of the retina and address vision loss in people with CLN2 disease.
How is TTX-181 Designed to Work?
Current treatment for the neurological effects of CLN2 disease involves biweekly intracerebroventricular (ICV) enzyme replacement therapy (ERT) with a human recombinant form of TPP1, cerliponase alfa, via an indwelling port. While ERT has been shown to slow progression of motor and language loss, it does not stop or reverse most manifestations of the disease. Tern Therapeutics is developing an investigational gene therapy, TTX-181, designed to deliver a working copy of the TPP1 gene to the brain via the cerebrospinal fluid (CSF), so that it can make an ongoing supply of TPP1 enzyme to prevent worsening of neurological degeneration in people with CLN2 disease.
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