CDMO Andelyn Biosciences partnered with Tern Therapeutics (Tern) to perform late-stage process performance qualification (PPQ) manufacturing of Tern’s TTX-381 program. The bioproduct is an investigational gene therapy targeting vision loss in children with CLN2 Batten disease, a rare pediatric neuro-degenerative disorder with no available treatment for the ocular manifestations of the disease.

‍CLN2, a form of Batten disease caused by TPP1 protein deficiency, is a pediatric neurodegenerative disorder with an estimated incidence of 0.5 per 100,000 live births. Most affected children begin experiencing seizures and language, motor, and cognitive decline between ages two and four, with vision loss beginning soon afterwards that rapidly leads to total loss of vision as early as age seven.

TTX-381 is a one-time, subretinal AAV gene therapy that is designed to deliver a functional copy of the TPP1 gene directly to the retinal cells to produce the missing TPP1 enzyme and preserve patient vision, with the potential to profoundly improve patient quality of life and independence.

“Partnering with Tern Therapeutics on this transformative program underscores Andelyn’s commitment to enabling breakthrough therapies for rare and devastating diseases,” notes Matt Niloff, CCO at Andelyn, “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.”

“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,” adds Alex Bailey, CEO at Tern.