Columbus, Ohio, August 18, 2026
Andelyn Biosciences, Inc., a patient-focused cell and gene therapy contract development and manufacturing organization (CDMO), has partnered with Queen’s University to develop and manufacture AAV9-GM2, an investigational gene therapy candidate for GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases. The collaboration represents an important manufacturing milestone for a rare-disease gene therapy program and highlights the growing role of specialized CDMOs in advancing AAV-based therapies from development toward clinical testing. Under the agreement, Andelyn will use its AAV Curator® Platform to support a multi-phase development and manufacturing program designed to prepare AAV9-GM2 for future patient dosing.
Advancing Gene Therapy for GM2 Gangliosidoses
GM2 gangliosidoses are rare inherited disorders in which genetic mutations interfere with normal lysosomal function, causing harmful substances to accumulate inside cells. The resulting neurological damage can progressively affect the brain and spinal cord, with severe forms often appearing during infancy and other forms emerging later in childhood, adolescence, or adulthood. Tay-Sachs and Sandhoff diseases are among the best-known forms of GM2 gangliosidosis, for which there remains a significant need for effective therapeutic options. The development of AAV9-GM2 reflects efforts to address the underlying genetic cause of these devastating disorders through gene therapy, an advanced therapeutic approach designed to deliver genetic material to affected cells. By supporting vector development and manufacturing, Andelyn will contribute specialized capabilities needed to advance the program toward clinical evaluation.
Andelyn Applies Its AAV Curator® Manufacturing Platform
As part of the collaboration, Andelyn Biosciences will leverage its AAV Curator® Platform, a regulatory-proven viral vector development and manufacturing platform designed to provide a consistent and adaptable approach to gene therapy production. The platform includes an established cell line and a modular strategy that allows manufacturing unit operations to be adapted according to individual program requirements. Andelyn says the approach is intended to support both yield and product quality, two critical considerations for the development of clinical-grade viral vectors. The company brings more than 20 years of experience in cell and gene therapy manufacturing and has produced cGMP material for more than 500 clinical batches and 85 global clinical trials. Its capabilities span plasmid engineering, process and analytical development, viral vector manufacturing, characterization, and cGMP clinical and commercial production. The company also operates configurable platforms supporting both suspension and adherent manufacturing processes.
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Strategic Collaboration Supports Rare-Disease Development
The partnership with Queen’s University underscores the importance of specialized manufacturing expertise in advancing therapies for rare and ultra-rare diseases. According to the companies, Andelyn’s manufacturing experience and quality systems are expected to help support the upcoming GM2 program and provide a reliable supply of viral vector material for clinical development. Queen’s University researchers, including medical geneticist and Professor of Pediatrics Dr. Jagdeep Walia, are involved in the program, bringing clinical and genetic-disease expertise to the collaboration. The initiative also demonstrates how academic research programs and specialized biotechnology CDMOs can work together to move promising gene therapy candidates closer to clinical testing. For patients and families affected by GM2 gangliosidoses, continued progress in gene therapy research, viral vector manufacturing, and cGMP production could help accelerate the development of new treatment approaches. The Andelyn–Queen’s University collaboration therefore represents a notable development in the broader cell and gene therapy manufacturing ecosystem, particularly for rare neurological diseases where scalable, high-quality AAV production is essential for clinical advancement.
Source: Andelyn Biosciences press relese
