Grant Award Details
- To achieve an active IND for clinical testing of autologous blood stem cells edited to restore iduronidase expression in Hurler's Syndrome or MPSI patients.
Grant Application Details
- Genome Editing of Autologous Hematopoietic Stem Cells to Treat Severe Mucopolysaccharidosis type 1 (Hurler Syndrome)
Therapeutic Candidate or Device
Autologous blood stem cells edited to restore iduronidase expression
Severe Mucopolysaccharidosis Type 1 (MPS1/ Hurler's syndrome)
Autologous blood stem cells undergo genome editing to restore the production of the missing enzyme. These cells are returned to the patient to replace their bone marrow, where they can secrete functional enzyme and become a permanent enzyme depot. The bone marrow also generates cells that migrate into affected organs, including the brain, to deliver the enzyme locally and prevent neurodegeneration and visceral and skeletal disease.
Unmet Medical Need
Severe MPS1 is a progressive disease without effective treatment. Allogeneic hematopoietic stem cell transplantation is used, but it is limited by the need for matched donors (causing delay/progression), insufficient disease correction, and the risks for graft-versus-host disease and graft failure
Filing of IND application with the FDA
Major Proposed Activities
- Develop process for Patient Scale Manufacturing Runs and Complete three that meet release specifications
- Completion of nonclinical safety studies
- Submit clinical protocol to IRB and file an IND with the FDA
For babies with MPS1, early detection and proper treatment can prevent severe disease manifestations, including neurodegeneration. Accordingly, California is one of thirty three states that screens every newborn for MPS1, and it is estimated to have the highest number of patients. Autologous transplantation of genome-edited cells is a safer, more effective therapy for MPS1. Investigators and patients in California have a unique opportunity to establish a definitive therapy for this disease