Biotech companies partner on Batten disease gene therapy
Investigational treatment TTX-381 to target vision loss in children
Tern Therapeutics is partnering with Andelyn Biosciences to develop its investigational gene therapy that targets vision loss in children with CLN2 Batten disease, also known as late-infantile Batten disease.
Andelyn, a contract development and manufacturing organization specializing in cell and gene therapy, will participate in the late-stage process performance qualification (PPQ) phase for TTX-381. PPQ is a validation step to demonstrate the ability to produce gene therapy that adheres to high-quality, predefined standards.
“Our deep experience in AAV process development and manufacturing, coupled with regulatory alignment and proven execution of [quality-consistent] batches, positions us to help bring this therapy to patients as efficiently and safely as possible,” Matt Niloff, chief commercial officer at Andelyn, said in a company press release.
CLN2 Batten disease is caused by mutations in the TPP1 gene that reduce or halt the production or function of tripeptidyl peptidase 1, an enzyme needed to break down the lipofuscin molecule. The toxic buildup of this molecule inside cells results in TPP1 enzyme defects and disease symptoms. Most affected children begin having seizures and motor and cognitive declines between ages 2 and 4, and total loss of vision as early as age 7.
What is TTX-381?
TTX-381 targets vision loss in children with CLN2 Batten disease, for which no available treatments exist. It uses a harmless adeno-associated virus (AAV) to deliver a functional version of the TPP1 gene to cells in the retina, the light-sensitive layer in the back of the eye where images are formed. The production of the missing enzyme is expected to preserve patients’ vision and improve their quality of life and independence.
Andelyn said the collaboration demonstrates its ability to accelerate gene therapy production from preclinical to commercial stages, leveraging its AAV manufacturing, state-of-the-art facilities, and patient-first approach.
“We’re excited to partner with Andelyn and leverage their long history with AAV technologies, strong late-stage manufacturing capability, and a shared dedication to rare disease patients,” said Alex Bailey, CEO at Tern. “Together, we aim to deliver a therapy that has the potential to change the lives of children and families facing CLN2 Batten disease, providing hope where today there are no effective treatment options.”
TTX-381 is being tested in a Phase 1/2 trial (NCT05791864), which may still enroll children with CLN2 Batten disease at a site in the U.K. The study aims to include 16 children, ages 1 to 7, who receive biweekly infusions of the enzyme replacement therapy Brineura (cerliponase alfa). In the study, participants received a single injection of one of two TTX-381 dose levels in one eye, with the untreated eye serving as a control. The primary objective is to evaluate TTX-381’s safety and tolerability. Secondary goals include its efficacy at increasing TPP1 enzyme levels and reducing retinal degeneration. The participants will be followed for five years after TTX-381 is administered. The trial should be completed in 2030.
Tern is also developing TTX-181, a gene therapy that delivers a working version of TPP1 to the brain to prevent worsening neurodegeneration in CLN2 disease.
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