Andelyn Biosciences, a leading cell and gene therapy CDMO, has partnered with Tern Therapeutics to perform late-stage Process Performance Qualification (PPQ) manufacturing for Tern’s investigational gene therapy, TTX-381. The therapy targets vision loss in children with CLN2 Batten disease, an ultra-rare neurodegenerative disorder with no current treatment for ocular manifestations.
Science Significance
TTX-381 is a one-time subretinal AAV gene therapy designed to deliver a functional copy of the TPP1 gene directly to retinal cells. By restoring TPP1 enzyme activity, the therapy aims to preserve vision and slow disease progression. This approach represents a cutting-edge application of gene therapy for pediatric neurodegeneration, addressing the genetic cause of CLN2 while offering the potential to improve clinical outcomes for a condition with severe, life-altering manifestations.
Regulatory Significance
Late-stage PPQ manufacturing is a critical regulatory milestone, providing data required to demonstrate consistent, cGMP-compliant production of clinical-grade material. Successful execution supports future FDA submissions, including a Biologics License Application (BLA), and ensures that the therapy meets U.S. and global regulatory standards for safety, quality, and reproducibility prior to commercial launch.
Business Significance
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For Andelyn Biosciences, the collaboration reinforces its reputation as a premier CDMO for gene therapy, leveraging expertise in both suspension and adherent AAV manufacturing, advanced process development, and regulatory alignment. For Tern Therapeutics, partnering with Andelyn ensures a reliable and experienced manufacturing pathway for TTX-381, accelerating development timelines for a potentially first-in-class therapy in an ultra-rare disease space.
Patients’ Significance
Children affected by CLN2 Batten disease experience rapid cognitive, motor, and visual decline, with total vision loss often occurring by age seven. TTX-381 has the potential to preserve sight and quality of life, offering hope to patients and families facing a condition with no approved ocular treatments. By advancing late-stage manufacturing, this partnership brings patients closer to accessing a therapy that could transform disease trajectory and independence.
Policy Significance
This collaboration aligns with global incentives for rare pediatric diseases, including potential Orphan Drug, RMAT, and Priority Review Voucher programs. It exemplifies the application of regulatory frameworks designed to accelerate development and commercialization of therapies for high unmet medical needs, particularly in ultra-rare pediatric neurodegenerative disorders.
Transaction Highlights
Andelyn Biosciences and Tern Therapeutics have joined forces to execute late-stage PPQ manufacturing of TTX-381, a one-time subretinal AAV gene therapy for vision loss in CLN2 Batten disease. The partnership leverages Andelyn’s AAV manufacturing expertise, cGMP process compliance, and regulatory experience, while enabling Tern to advance a potentially transformative therapy for an ultra-rare pediatric population. This collaboration represents a strategic milestone in moving TTX-381 toward clinical and eventual commercial readiness.
Source: Andelyn Biosciences Press Release
