Andelyn Biosciences and Queen’s University Collaborate on Gene Therapy for Devastating Neurological Disorders
COLUMBUS, OH & KINGSTON, ONTARIO – [Date of Publication] – Andelyn Biosciences Inc., a leading patient-focused Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy, has announced a significant partnership with Queen’s University. This collaboration aims to accelerate the development and manufacturing of AAV9-GM2, a promising gene therapy candidate designed to treat GM2 gangliosidoses, a group of severe and often fatal neurological disorders including Tay-Sachs and Sandhoff diseases.
GM2 gangliosidoses are rare, inherited conditions characterized by the progressive destruction of nerve cells in the brain and spinal cord. As a type of lysosomal storage disorder (LSD), these diseases stem from genetic mutations that impair normal lysosomal function, leading to the toxic accumulation of specific fatty substances (gangliosides) within cells. This accumulation causes severe and irreversible neurological decline, with symptoms varying depending on the form, which can manifest from infancy through adulthood. The most common and devastating form typically presents in infancy.
Under the terms of this strategic partnership, Andelyn Biosciences will leverage its proprietary AAV Curator Platform. This robust platform will be instrumental in executing a multi-phase program to advance AAV9-GM2, bringing it closer to clinical trials and ultimately, to patients in need. The AAV Curator Platform is renowned for its ability to optimize AAV vector development and production, ensuring high-quality and scalable manufacturing processes essential for gene therapy applications.
Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed his enthusiasm for the collaboration: “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. 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, a distinguished medical geneticist and Professor in the Department of Pediatrics at Queen’s University, underscored the strategic importance of the partnership. "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," stated Dr. Walia. This emphasis on safety and quality is paramount in the development of gene therapies, particularly for vulnerable patient populations.
This latest announcement follows a period of significant growth and innovation for Andelyn Biosciences. Earlier this year, the company expanded its capabilities by launching a standardized lentiviral vector manufacturing platform, further solidifying its position as a comprehensive CDMO in the advanced therapy space.
The collaboration between Andelyn Biosciences and Queen’s University represents a crucial step forward in the fight against GM2 gangliosidoses, offering a beacon of hope for patients and their families by combining cutting-edge scientific research with advanced manufacturing expertise in the field of gene therapy. This partnership aims to transform the lives of those affected by these devastating neurological conditions. For more information on this significant collaboration, read the full announcement here: Andelyn Biosciences Partners with Queen’s University on GM2 Gene Therapy.
