NIB is honored to support development of Axovant's AXO-AAV-GM2 gene therapy
On Nov. 09 Axovant Gene Therapies received FDAs clearance on Investigational New Drug (IND) Application to initiate a registrational study of AXO-AAV-GM2 gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease (https://bit.ly/3n5G5yt).
National institute of Biology (NIB) serves as a contract lab for Axovant Gene Therapies, providing time-effective development and consultation on specific analytical techniques, such as droplet digital polymerase chain reaction (ddPCR), required for accurate characterization of the drug product.
NIB is honored to be a part of this milestone and is grateful for acknowledgement received from Axovant's Director of Program Managment, Ira Liolis, who said, “Thank you for your excellent work and amazing support toward advancing the program and we look forward to continuing the collaboration.”
AXO-AAV-GM2 is an investigational gene therapy for Tay-Sachs and Sandhoff diseases, which are rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode β-Hexosaminidase A, HEXA and HEXB. Children affected by Tay-Sachs and Sandhoff diseases suffer from a progressively debilitating disease course and reduced life expectancy.
About Axovant Gene Therapies
Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinson’s disease.
Effective today (November 13) Axovant is now renamed to Sio Gene Therapies.
Department of Biotechnology and Systems Biology at the National Institute of Biology is holder of worldwide recognized expertise in the field of virus diagnostics, characterization and quantification.