👁️ This investigational gene therapy delivers the missing TPP1 gene to retinal cells. The goal is to improve quality of life for affected children.
🚀 Both companies are committed to advancing crucial treatments for rare diseases.
Introduction:
This article discusses the collaboration between Andelyn Biosciences and Tern Therapeutics aimed at advancing gene therapy manufacturing for Tern’s TTX-381 program, a promising treatment for CLN2 Batten disease. This partnership represents a significant step towards providing hope to children suffering from a debilitating genetic disorder that currently has no effective treatment.
- Andelyn Biosciences has partnered with Tern Therapeutics for the late-stage process performance qualification (PPQ) manufacturing of TTX-381, a gene therapy targeting vision loss.
- CLN2 Batten disease, a rare pediatric neurodegenerative condition, arises from TPP1 protein deficiency and typically leads to severe vision loss in affected children.
- TTX-381 is designed as a one-time, subretinal AAV gene therapy that aims to deliver a functional TPP1 gene directly to retinal cells, enabling the production of the missing enzyme and potentially preserving vision.
- Both companies emphasize their commitment to transforming the lives of children and families impacted by CLN2 Batten disease through this collaboration.
- Andelyn’s expertise in AAV process development and manufacturing, combined with Tern’s innovative therapeutic approach, positions the partners to deliver this important treatment efficiently and safely.
Conclusion:
The partnership between Andelyn Biosciences and Tern Therapeutics is a hopeful advance in the quest to provide effective treatment options for CLN2 Batten disease, underscoring the potential of gene therapy in addressing rare genetic disorders. As manufacturing progresses, this collaboration may pave the way for new therapeutic avenues that can significantly improve the quality of life for affected patients.
