Andelyn and Amplo Partner to Scale AAV Manufacturing for NMJ Disorders

Andelyn Biosciences, Inc., a leading cell and gene therapy contract development and manufacturing organisation (CDMO), has entered into a partnership with Amplo Biotechnology to accelerate the production of clinical-grade adeno-associated virus (AAV) material for therapies targeting neuromuscular junction (NMJ) disorders. The collaboration will leverage Andelyn’s proprietary suspension AAV Curator® platform, a scalable system designed to optimise AAV vector production and streamline the path to cGMP and commercial manufacturing.

NMJ disorders are a group of rare but serious conditions caused by genetic mutations affecting the junctions between nerves and muscles. These disruptions impair signal transmission, leading to progressive muscle weakness that, in severe cases, can necessitate respiratory support, tube feeding, or the use of wheelchairs. While these diseases are often diagnosed in childhood, symptoms can also present later in life, creating challenges for timely diagnosis and treatment.

Gene therapy has emerged as a promising approach to NMJ conditions because AAV vectors can deliver functional copies of faulty genes directly to affected tissues, potentially addressing the root cause of the disease rather than just managing symptoms. However, the complexity and cost of manufacturing high-quality viral vectors at a clinical scale have historically slowed the translation of these therapies into trials.

Andelyn’s Curator® platform aims to address that bottleneck. According to Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, “Andelyn Biosciences suspension AAV Curator platform is a highly characterised, scalable suspension AAV platform that leverages a data-driven approach to adapt and optimise customer processes while providing a pathway to cGMP and commercial manufacturing. 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.”

For Amplo Biotechnology, the partnership represents an opportunity to move its preclinical pipeline closer to clinical evaluation. The company focuses on regenerative AAV medicines for NMJ-affecting conditions, a category that includes diseases such as congenital myasthenic syndromes and Lambert-Eaton myasthenic syndrome. In a statement, the company said the collaboration “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.”

Based in Columbus, Ohio, Andelyn Biosciences has become a prominent player in the gene therapy manufacturing ecosystem, known for its expertise in viral vector development and large-scale production. The company emphasises that partnerships like this are critical to advancing gene therapy from discovery into the clinic, noting that “by collaborating with leading organisations such as Amplo Biotechnology, Andelyn is at the forefront of advancing life-changing gene therapies… and transforming the landscape of healthcare through the development of groundbreaking treatments.”

The partnership underscores a growing trend in the biotechnology sector: specialised CDMOs and small, innovative biotech companies joining forces to overcome the manufacturing and regulatory hurdles of gene therapy. For NMJ disorders, where effective treatments are scarce, this collaboration could accelerate the path toward much-needed clinical trials and, ultimately, patient access.