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AAV9-GM2
- Development stage
- Preclinical
- Lead developer
- Queen's University
- Modality
- Gene Therapies
- Administration
- Parenteral
01
Overview
AAV9-GM2 is an investigational gene therapy being developed through a partnership between Queen's University and Andelyn Biosciences. It is designed to treat GM2 gangliosidoses, a group of rare, inherited lysosomal storage disorders including Tay-Sachs disease and Sandhoff disease. The therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver functional copies of the genes (HEXA and HEXB) that encode the hexosaminidase A enzyme. Deficiency of this enzyme leads to the toxic accumulation of GM2 gangliosides in the brain and spinal cord, causing progressive neurodegeneration. AAV9-GM2 aims to restore enzyme activity and is currently in the research and development stage, utilizing Andelyn's AAV Curator Platform for manufacturing.
- Other names
- AAV9-GM2 gene therapyAAV-9-GM2 gene therapyAAV 9-GM2 gene therapy
02
Targets
Beta-hexosaminidase A (Hex A)GM2 (Ganglioside GM2)
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