CDMO Andelyn Biosciences partnered with Amplo Biotechnology, which focuses on adeno-associated virus (AAV) regenerative medicines for neuromuscular junction-(NMJ) affecting conditions, to provide scalable manufacturing of clinical-grade AAV material, leveraging Andelyn’s suspension AAV Curator® platform.
Diseases of the NMJ typically result in muscle weakness with severe cases requiring the use of respiratory support, the use of wheelchair for ambulation, and/or tube feeding. In some cases, NMJ disorders can be life-threatening. Genetic NMJ diseases are typically diagnosed in early childhood but can also manifest in adolescence or adulthood.
“Andelyn Biosciences suspension AAV Curator platform is a highly characterized, scalable suspension AAV platform that leverages a data-driven approach to adapt and optimize customer processes while providing a pathway to cGMP and commercial manufacturing, efforts to bring hope to patients affected by debilitating NMJ diseases,” according to Matt Niloff, chief commercial officer at Andelyn.
By adopting this platform, Amplo Biotechnology aims to advance its AAV gene therapy programs toward clinical evaluation, focusing on the safety and efficacy required for upcoming trials.
“Our Curator platform and end-to-end capabilities are designed to help partners achieve key development and clinical milestones,” adds Niloff.
The post Andelyn and Amplo Biotechnology Partner to Manufacture Gene Therapies for NMJ Diseases appeared first on GEN – Genetic Engineering and Biotechnology News.