Andelyn Biosciences Partners with Queen's University for Gene Therapy Development

Collaboration for Gene Therapy Development

Andelyn Biosciences, a prominent cell and gene therapy Contract Development and Manufacturing Organization (CDMO), is embarking on an exciting partnership with Queen's University to develop an innovative gene therapy, AAV9-GM2, targeted towards the treatment of GM2 gangliosidoses. This condition includes devastating diseases such as Tay-Sachs and Sandhoff disease, which progressively degrade nerve cells in individuals due to genetic mutations.

Understanding GM2 Gangliosidoses

GM2 gangliosidosis is a rare disorder caused by inheritable genetic mutations that lead to the malfunction of lysosomes. This results in the harmful accumulation of substances in cells, ultimately resulting in severe neurological decline. The most prevalent form of GM2 typically emerges in infancy, but there are variants that may appear in childhood, adolescence, or even adulthood. This highlights the urgent need for effective gene therapy options for individuals affected by these conditions and their families.

The AAV9-GM2 Gene Therapy Candidate

Under this strategic collaboration, Andelyn Biosciences will leverage its AAV Curator® Platform to execute a multi-phase development strategy for the AAV9-GM2 gene therapy. The AAV Curator® Platform is recognized for its proven regulatory track record and employs an adaptable approach to meet the specific needs of complex gene therapy programs.

The ability to deliver high yields without compromising quality makes this platform particularly valuable. As Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, articulates, the commitment lies in enhancing patient outcomes through innovative therapeutic technologies, enabling those struggling with GM2 gangliosidoses to hope for a better future.

Clinical Trial and Manufacturing Quality

Dr. Jagdeep Walia, a medical geneticist and a professor at Queen's University, remarks on the strategic importance of this partnership. He emphasizes Andelyn's established credentials in producing viral vectors for various gene therapy initiatives. This collaboration assures that the clinical trial participants will receive a high-quality and safe gene therapy product, which is paramount for effective treatment.

As Andelyn Biosciences invests its expertise into this partnership, it reinforces its dedication to supporting innovative gene therapies aimed at treating rare and ultra-rare diseases. The efficient pace of development, alongside exceptional quality and manufacturing capabilities, is critical in bringing these life-enhancing therapies closer to patients who need them most.

About Andelyn Biosciences

Andelyn Biosciences has built a solid reputation as a full-service CDMO specializing in the development and production of viral vectors for gene therapy. With over two decades of experience, the firm has supplied more than 500 clinical batches and supported 85 global clinical trials. Situated in Columbus, Ohio, Andelyn is equipped to take client projects from the initial concept stage through to GMP-compliant manufacturing, emphasizing quality and innovation at every stage of development.

With the versatile and data-driven Curator® Cell and Gene Therapy Platforms, Andelyn can expedite programs and produce high-quality outputs. They possess the requisite clinical manufacturing capabilities to bring advanced therapies to market efficiently.

For more details on Andelyn Biosciences and its revolutionary gene therapy initiatives, visit their official website or follow them on their professional LinkedIn page.

About Queen's University

Queen's University has long been a leader in research and innovation, striving to address global challenges through scholarly work. With a vibrant community of over 28,000 students, Queen's fosters an enriching academic environment driven by diverse perspectives. In 2026, the university’s commitment to impactful research earned it the distinction of ranking fourth globally in the Times Higher Education Impact Rankings, underscoring its contribution to the United Nations' Sustainable Development Goals.

Through this partnership with Andelyn Biosciences, Queen's University solidifies its role in advancing ground-breaking research and innovative therapies that make a difference in patients' lives worldwide.