Andelyn Biosciences Partners with Queen's University on GM2 Gangliosidosis Gene Therapy

Andelyn Biosciences announced on August 18, 2026 that Canada’s Queen’s University has selected the Ohio-based cell and gene therapy contract development and manufacturing organization to develop and manufacture AAV9-GM2, an adeno-associated virus (AAV) gene therapy candidate for GM2 gangliosidoses.¹

The disease category includes Tay-Sachs disease and Sandhoff disease, which are both rare inherited disorders classified as lysosomal storage diseases that progressively damage nerve cells in the brain and spinal cord. Symptoms can appear in infancy, childhood, adolescence, or adulthood, depending on the degree of enzyme deficiency involved.

How Will the Vector Be Manufactured?

The program will run through Andelyn's AAV Curator platform, which is a viral vector process built around a proprietary cell line and a modular approach to adapting individual unit operations for different programs. It is structured as a multi-phase development effort; neither organization has disclosed a timeline, contract value, or specific manufacturing milestones.

"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, in the press release.¹ “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.”

What Causes GM2 Gangliosidosis, and Why Target It with Gene Therapy?

GM2 gangliosidosis results from inherited mutations that impair the body’s ability to break down certain fatty substances within nerve cells, allowing them to accumulate to toxic levels over time. Because the disorder traces back to a single defective gene, it has become a target for gene therapy approaches designed to deliver a functional copy of that gene directly to affected tissue rather than manage symptoms after the fact. The AAV9 vector selected for this program is frequently used in gene therapies aimed at the central nervous system because of its capacity to cross the blood-brain barrier.

“This partnership with Andelyn is very strategic as they have the established expertise of producing vectors for many gene therapy programs,” said Dr. Jagdeep Walia, a medical geneticist and professor in the Department of Pediatrics at Queen's University, in a press release.¹ “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.”

Andelyn’s Other Curator Platform

The AAV Curator is not Andelyn’s only platform. In May 2026, the company announced the launch of its LVV Curator Platform, a standardized lentiviral vector (LVV) manufacturing tool built on the modular approach of its AAV platform.² The LVV Curator is designed to support early-phase programs with a path to scale-up in the future.

“By combining a proven platform foundation with a flexible, partnership-driven business model, we are helping innovators bridge the gap from discovery to clinical manufacturing more efficiently,” Niloff said in the press release.²

In April of 2025, Andrew Moreo, head of Process Development and Pre-clinical Manufacturing at Andelyn Biosciences, spoke with PharmTech’s sister publication, BioPharm International about the company’s curator platform.

The platform has “a modular design with optimization actually built into the process itself, into the platform itself, that makes things go much smoother,” Moreo explained.³ “And in our business, smooth is fast. As not every product is created equal, we have to be able to provide the consistency and efficiency of a platform, but at the same time, also provide flexibility because different genes, approaches, vectors, serotypes, and transgenes perform differently and have different demands based on the needs of the indication.”

References

1. Andelyn Biosciences to implement Curator Platform to develop and manufacture gene therapy AAV9-GM2 for Queen’s University to treat GM2 gangliosidoses. Press release. Andelyn Biosciences. August 18, 2026. https://www.andelynbio.com/news-media/andelyn-biosciences-to-develop-and-manufacture-gene-therapy-aav9-gm2-for-queens-university-to-treat-gm2-gangliosidoses
2. Andelyn Biosciences launches LVV Curator Platform, bringing proven viral vector platform pedigree to lentiviral vector manufacturing for cell therapies. Press release. Andelyn Biosciences. May 11, 2025. https://www.andelynbio.com/news-media/andelyn-biosciences-launches-lvv-curator-platform
3. Haigney S. Novel Oncology Biotherapeutics: Gene Therapies. BioPharm International. April 10, 2025. https://www.biopharminternational.com/view/novel-oncology-biotherapeutics-gene-therapies