STEM Technology
STEMs (Stem cells engineered into micropharmacies) are harvested from bone marrow and genetically engineered to provide durable cell therapy. Clinical safety and probable effectiveness of our STEM therapy has been demonstrated in Fabry disease (PMID 39794302).
Fabry STEM Program (GLA: alpha-galactosidase A):
Treating Fabry disease caused by GLA deficiency - a condition affecting over 800,000 persons worldwide.
Pompe STEM Program (GAA: acid maltase):
Treating Pompe disease caused by GAA deficiency - a condition affecting over 400,000 persons worldwide.
Gaucher STEM Program (GLB1: acid glucosylceramidase):
Treating Gaucher disease caused by GLB1 deficiency - a condition affecting over 160,000 persons worldwide.
Our Programs using STEM technology provide a cell-based gene therapy that uses tissue harvested from the patient bone marrow (autologous-derived). The STEM procedure involves mobilizing a patient’s hematopoietic stem cells (HSCs) and transporting them to a GMP lab for gene augmentation using a lentiviral vector. The introduced gene creates high levels of secreted enzyme, which is taken up by neighboring cells and allows normal enzyme function to be restored in the patient.
STEM Therapy
The STEM procedure involves induction of HSC mobilization in the patient.
Mobilized cells are then harvested from the patient by apheresis.
In the lab the HSCs are isolated by CD34+ selection.
Lentivector transduction of CD34+ cells is performed.
The resulting cells contain a working copy of the target gene.
The modified HSCs are reintroduced into the patient as a transplant procedure where engraftment into the bone marrow ensues.
The resulting modified blood cells secrete the normal enzyme which leads to restoration of gene function.
Our STEM therapy approach is an improvement over intermittent enzyme exposure with recombinant protein transfusion. Unlike traditional enzyme replacement therapy (ERT), our STEM therapy provides uninterrupted cross-correction - the patient obtains a more continuous supply of the replacement enzyme lasting for at least 5 years in a Canadian clinical trial (PMID 39794302). Furthermore, the transplant leukocytes are known spread systemically to a wide variety of compartments in the body, including the brain, thus the blood brain barrier problem with traditional recombinant protein transfusions is a much lower obstacle. As a result, successful treatment of neurological complication of enzyme deficiency can be expected.
