Andelyn Biosciences and Amplo Biotechnology Partner to Manufacture Novel Gene Therapies for Diseases Affecting the Neuromuscular Junction

COLUMBUS, Ohio, Aug. 21, 2025 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Amplo Biotechnology, a pioneer in adeno-associated virus (AAV) regenerative medicines for neuromuscular junction (NMJ) affecting conditions. The partnership will 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. 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.

"As a leading clinical and commercial CDMO, we are honored 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.

Andelyn is enabling the progression of life-altering cell and gene therapies for both rare and prevalent diseases with the highest quality standards and scalable end-to-end development and manufacturing capabilities at its Columbus, Ohio facilities. By collaborating with leading organizations such as Amplo Biotechnology, Andelyn is at the forefront of advancing life-changing gene therapies. This partnership not only underscores Andelyn's commitment to innovation but also highlights its role in transforming the landscape of healthcare through the development of groundbreaking treatments.‍

About Andelyn Biosciences, Inc.

Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization, and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of its development and manufacturing facilities in Columbus, Ohio, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn can accelerate programs and deliver high-quality products by developing and manufacturing processes on its configurable, data-driven AAV Curator^® Platform or tech transfer in an established client program. Capabilities include cGMP manufacturing for suspension processes up to a 2,000-liter and adherent processes. A rigorous quality system, regulatory support, and supply chain vertical integration further advantages clients in bringing their critical therapies to market. For more information, visit andelynbio.com.

About Amplo Biotechnology, Inc.

Amplo Biotechnology is a late-stage preclinical company developing regenerative AAV gene therapies with an initial focus on restoring NMJ function in rare, genetically defined disorders. Amplo's lead program, AMP-101, is advancing toward a first-in-human trial in Dok7 congenital myasthenic syndrome, built on research from the University of Tokyo and Oxford.

SOURCE Andelyn Biosciences