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Sio Gene Therapies announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase I/II trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases. Miguel Sena-Esteves, PhD, is a principal scientist of the research program at UMMS.

Thomas Feldborg and Daria Rokina, of Denmark, chose to enroll their baby daughter, Alissa, in an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases, to “let hope conquer the fears,” according to a report in USA Today chronicling their journey.

Oguz Cataltepe, MD, describes the innovative neurosurgical procedure used to treat patients in the new clinical trial for a gene therapy targeting infantile Tay-Sachs and Sandhoff diseases.

UMass Medical School scientist Miguel Sena-Esteves, PhD, and colleagues are nearing human clinical trials on a gene therapy for Tay-Sachs and Sandhoff diseases.

Axovant Sciences, a Swiss company developing gene therapies for neurological diseases, has licensed exclusive worldwide rights for the development and commercialization of two novel gene therapy programs for Tay-Sachs and Sandhoff diseases from UMass Medical School.

Medical School research discoveries, has announced that the FDA has lifted its clinical hold and cleared Axovant’s Investigational New Drug application to initiate a registrational study of gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease.

licensed exclusive worldwide rights from UMass Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases. Research into potential therapies for lysosomal storage diseases such as Tay-Sachs, Sandhoff disease and GM1 gangliosidosis at UMass Medical School and Auburn University has led to significant advances in the field. Miguel Sena-Esteves, PhD, associate professor of neurology at UMass Medical

including Tay-Sachs and Sandhoff diseases. GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene that cause impaired production of the β-galactosidase enzyme. Currently, there are no FDA-approved treatment options for GM1 gangliosidosis. Miguel Sena-Esteves, PhD, and Heather Gray-Edwards, DVM, PhD Research into the causes and potential therapies for lysosomal storage diseases such as Tay-Sachs and Sandhoff diseases and GM1 gangliosidosis

research from the Blu Genes Foundation will allow us to take more than a decade of scientific discovery into the clinic, where our novel gene therapy approach will directly impact patient lives,” Dr. Gray-Edwards said. Tay-Sachs, and a similar disease, Sandhoff disease, are inherited neurologic diseases that occur when genetic mutations prevent cells from producing enzymes needed to break down and recycle materials. Without these enzymes, the materials accumulate to toxic levels, slowly destroying the nervous

Auburn University College of Veterinary Medicine, have worked collaboratively for nearly 15 years, combining small and large animal studies to cure rare diseases. Research into potential therapies for lysosomal storage diseases such as Tay-Sachs, Sandhoff disease and GM1 gangliosidosis at UMass Medical School has already led to significant advances in the field, including research and development of the gene therapy vector used to deliver functioning copies of the defective genes that cause disease