- Andelyn Biosciences will perform late-stage Process Performance Qualification (PPQ) manufacturing for Tern Therapeutics’ investigational gene therapy TTX-381.
- TTX-381 targets vision loss in children with CLN2 Batten disease, an ultra-rare paediatric neurodegenerative disorder with no available treatment.
Andelyn Biosciences, Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Tern Therapeutics to carry out late-stage Process Performance Qualification (PPQ) manufacturing of Tern’s TTX-381 programme. The investigational therapy targets vision loss in children with CLN2 Batten disease, a rare paediatric neurodegenerative disorder.
CLN2 Batten disease results from TPP1 protein deficiency and affects around 0.5 per 100,000 live births. Children typically experience seizures and decline in motor, language, and cognitive abilities between ages two and four. Vision loss follows soon after, often progressing to complete blindness by age seven.
TTX-381 is a one-time subretinal adeno-associated virus (AAV) gene therapy. It is designed to deliver a functional copy of the TPP1 gene directly to retinal cells to restore enzyme production and preserve vision. The therapy is intended to improve quality of life and independence for affected children.
Matt Niloff, Chief Commercial Officer at Andelyn, said: “Partnering with Tern Therapeutics on this transformative programme underscores Andelyn’s commitment to enabling breakthrough therapies for rare and devastating diseases.”
The collaboration builds on Andelyn’s experience in AAV vector manufacturing and PPQ batch production. It also aligns with the company’s broader mission to accelerate gene therapies from preclinical stages to commercial readiness, supported by its facilities and expertise in suspension and adherent AAV manufacture.
