Tern Therapeutics is at the forefront of developing innovative gene therapies aimed at treating rare diseases, particularly focusing on CLN2 disease, a form of Batten disease. Their lead candidate, TTX-381, is designed to address the ocular manifestations of CLN2 by delivering a functional copy of the TPP1 gene directly to the retina. This approach aims to enable the retina to produce the TPP1 enzyme, which is crucial for breaking down waste materials in cells, thereby potentially preventing vision loss associated with the disease. The clinical trial for TTX-381 is currently being conducted at Great Ormond Street Hospital in London, highlighting the urgency and commitment of Tern Therapeutics to provide transformative treatments for affected individuals.

In addition to TTX-381, Tern Therapeutics is also developing TTX-181, which targets the neurological aspects of CLN2 disease. This investigational gene therapy aims to deliver the TPP1 gene to the brain via the cerebrospinal fluid, allowing for a continuous supply of the enzyme to mitigate neurological degeneration. Current treatments, such as enzyme replacement therapy, have limitations in reversing the disease's effects, particularly in the eyes and brain. By focusing on gene therapy, Tern Therapeutics seeks to provide a more effective and lasting solution for patients suffering from the debilitating impacts of CLN2 disease.

United States

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, we are driven to deliver transformative treatments with urgency to those living around the world with rare disease.

Tern Therapeutics

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