Andelyn Biosciences and Amplo Partner on AAV Gene Therapy

Andelyn Biosciences, a contract development and manufacturing organization (CDMO), has formed a collaboration with Amplo Biotechnology to manufacture adeno-associated virus (AAV) gene therapies targeting conditions impacting the neuromuscular junction (NMJ). This partnership aims to enhance the scalable production of clinical-grade AAV materials, utilizing Andelyn’s innovative suspension AAV Curator platform.

Manufacturing Advancements for Gene Therapy

The collaboration is designed to support the transition from research to clinical applications by ensuring compliance with current good manufacturing practices (cGMP). Through this agreement, Amplo will utilize Andelyn’s platform to advance its AAV gene therapy initiatives, focusing on the critical aspects of efficacy and safety required for upcoming clinical evaluations.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed enthusiasm about the partnership, stating, “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. Our Curator platform and end-to-end capabilities are designed to help partners achieve key development and clinical milestones.”

Conditions affecting the NMJ often result in significant muscle weakness, which can escalate to severe complications requiring respiratory support, wheelchair dependence, or tube feeding. These disorders, typically diagnosed in early childhood, can also manifest during adolescence or adulthood and may be life-threatening.

Focus on Clinical Trials and Research

Based in Idaho, USA, Andelyn Biosciences specializes in characterizing, developing, and producing viral vectors for gene therapy applications. Amplo’s primary program, AMP-101, is progressing toward its first-in-human study for Dok7 congenital myasthenic syndrome. This research is rooted in studies conducted at the prestigious University of Tokyo and University of Oxford, highlighting the collaboration’s commitment to advancing scientific research.

Amplo previously secured funding from the National Institutes of Health’s National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIH-NIAMS) for its gene therapy program, AMP-201, reflecting the organization’s dedication to addressing challenging medical conditions through innovative therapies.

The collaboration between Andelyn Biosciences and Amplo Biotechnology represents a significant step forward in the development of gene therapies for NMJ diseases, offering hope to many patients facing these serious health challenges.