Andelyn Biosciences Partners with Queen's University to Manufacture AAV9-GM2 Gene Therapy for GM2 Gangliosidoses
核心洞察
Andelyn Biosciences (搜索) has partnered with Queen's University to develop and manufacture AAV9-GM2 (搜索), a gene therapy candidate for GM2 gangliosidoses including Tay-Sachs and Sandhoff diseases.
The collaboration leverages Andelyn's regulatory-proven AAV Curator® Platform to execute a multi-phase program advancing AAV9-GM2 (搜索) toward patient dosing.
GM2 gangliosidosis (搜索) is a rare, inherited lysosomal storage disorder that progressively destroys nerve cells in the brain and spinal cord, causing severe neurological decline.
Andelyn Biosciences (搜索), 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 (搜索), a gene therapy candidate for the treatment of GM2 gangliosidoses, such as Tay-Sachs and Sandhoff diseases. The announcement was made on August 18, 2026, from Columbus, Ohio.
GM2 gangliosidosis (搜索) is a rare, inherited disorder that progressively destroys nerve cells in the brain and spinal cord. While the most common form presents in infancy, other forms may emerge in childhood, adolescence, or adulthood. As a lysosomal storage disorder (LSD), GM2 is caused by genetic mutations that disrupt normal lysosomal function, leading to the accumulation of harmful substances in cells and severe neurological decline.
Under the partnership, Andelyn is leveraging its AAV Curator® Platform to execute a multi-phase program to advance AAV9-GM2 (搜索) for dosing patients. The AAV Curator® Platform is Andelyn's long-standing, regulatory-proven viral vector process, complete with a cell line and a modular approach to adapting unit operations to program requirements. With this methodology, both yield and quality are delivered.
"We are proud to partner with Queen's University to advance this gene therapy candidate for the benefit of patients and families facing the challenges of GM2 gangliosidoses," said Matt Niloff, Chief Commercial Officer at Andelyn Biosciences (搜索). "Our deep expertise in AAV development and production allows us to support Queen's University with the scale and quality rigor necessary to bring this life-changing therapy one step closer to reality."
Dr. Jagdeep Walia, medical geneticist and Professor in the Department of Pediatrics at Queen's University, said, "This partnership with Andelyn is very strategic as they have the established expertise of producing vectors for many gene therapy programs. Their track record and ability to manufacture the viral vector for the upcoming GM2 program gives us confidence that the participants in the clinical trial will be receiving the highest quality, safe product."
This partnership reflects Andelyn's continued commitment to supporting innovative gene therapy programs targeting rare and ultra-rare diseases, where speed, quality, and manufacturing expertise are critical to advancing therapies to patients.
Andelyn Biosciences (搜索) 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 500 clinical batches and 85 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 Curator® Cell and Gene Therapy Platforms, or by tech transfer within an established client program. Capabilities include cGMP manufacturing for suspension and adherent processes. A rigorous commercial-grade quality system, phase-appropriate manufacturing, and supply chain vertical integration further advantage clients in bringing their critical therapies to market.
Queen's University has a long history of scholarship, discovery, and innovation. Home to more than 28,000 students, Queen's offers a comprehensive research-intensive environment. In 2026, for the sixth year in a row, Queen's University has ranked in the top 10 globally of the Times Higher Education Impact Rankings, securing the position of 4th worldwide and 1st in Canada.