This therapy is aimed at addressing vision loss in children with this disease, a rare paediatric neurodegenerative disorder for which there is currently no treatment available for its ocular manifestations.
CLN2 Batten disease is caused by a deficiency in the TPP1 protein and has an estimated incidence rate of 0.5 per 100,000 live births.
TTX-381 is a subretinal adeno-associated virus (AAV) gene therapy specifically developed to deliver a functional TPP1 gene to retinal cells directly.
This approach aims to produce the TPP1 enzyme that is otherwise missing, thereby preserving vision.
Andelyn Biosciences will utilise its expertise in AAV vector manufacturing and PPQ batch production to facilitate the progression of TTX-381.
Andelyn chief commercial officer Matt Niloff said: “Partnering with Tern Therapeutics on this transformative programme underscores Andelyn’s commitment to enabling breakthrough therapies for rare and devastating diseases.
“Our deep experience in AAV process development and manufacturing, coupled with regulatory alignment and proven execution of PPQ batches, positions us to help bring this therapy to patients as efficiently and safely as possible.”
Tern CEO Alex Bailey said: “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.
“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.”