Andelyn Biosciences, a patient-focused cell and gene therapy contract development and manufacturing organisation (CDMO), has partnered with Amplo Biotechnology, a pioneer in adeno-associated virus (AAV) regenerative medicines for neuromuscular junction (NMJ)-affecting conditions.
The collaboration will enable the scalable production of clinical-grade AAV material.
Diseases of the NMJ typically result in muscle weakness with severe cases requiring the use of respiratory support, the use of a 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's AAV Curator is a highly characterised, scalable suspension platform that leverages a data-driven approach to adapt and optimise customer processes while providing a pathway to cGMP and commercial manufacturing.
By adopting this platform, Amplo Biotechnology aims to advance its AAV gene therapy programmes toward clinical evaluation, focusing on the safety and efficacy required for upcoming trials.
“As a leading clinical and commercial CDMO, we are honoured to support Amplo Biotechnology’s efforts to bring hope to patients affected by debilitating NMJ diseases,” said Matt Niloff, Chief Commercial Officer at Andelyn Biosciences.
“Our Curator platform and end-to-end capabilities are designed to help partners achieve key development and clinical milestones.”
Amplo Biotechnology stated that this partnership strengthens its ability to deliver high-quality AAV material and supports its mission to develop transformative therapies for families impacted by genetic NMJ disorders worldwide.
