Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM2 Gene Therapy in Patients with GM2 Gangliosidosis
- Designation complements previously granted Rare Pediatric Disease and Orphan Drug designations
- Enrollment ongoing in the registration-enabling trial of AXO-AAV-GM2 in Tay-Sachs/Sandhoff diseases
“The FDA’s decision to grant AXO-AAV-GM2 gene therapy Fast Track designation signifies an important milestone towards developing a safe and effective treatment for Tay-Sachs and Sandhoff diseases, both rare and fatal pediatric diseases with no approved treatments,” said
The current Phase 1/2 study (NCT04669535) is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal cord of pediatric participants with both infantile and juvenile GM2 gangliosidosis (also known as Tay-Sachs or Sandhoff disease).
GM2 gangliosidosis is a set of rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode the enzyme β-Hexosaminidase A. It can be categorized into two distinct diseases, Tay-Sachs disease, which results from a mutation in the gene encoding the alpha subunit of the β-Hexosaminidase A enzyme (HEXA), and Sandhoff disease, which results from a mutation in the gene encoding the beta subunit of the β-Hexosaminidase A enzyme (HEXB). Children affected by GM2 gangliosidosis suffer from a progressively debilitating disease course and reduced life expectancy. Currently, there are no FDA-approved treatment options for GM2 gangliosidosis.
AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the
AXO-AAV-GM2 has received Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation from the Food and Drug Administration and is the only gene therapy in clinical development for all pediatric forms of GM2 gangliosidosis.
In 2018, Sio licensed exclusive worldwide rights from UMass Chan Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.
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Source: Sio Gene Therapies