GLA-H Gene Therapy
GLA-H gene therapy uses genetically-modified hematopoietic stem cells (HSCs) to restore alpha-galactosidase A (a-gal A) enzyme activity in patients. This process involves modifying HSCs by a lentivector (LV) encoding an GLA gene, which enables the population of HSCs that are autologously sourced from the patient to be gene augmented to express high levels of a-gal A enzyme.
The 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 extra copies of the GLA gene.
The modified HSCs are reintroduced into the patient as a transplantation.
Engraftment into bone marrow ensues.
With GLA-H gene therapy, the differentiated progeny of the HSCs become the modified leukocyte population that spreads throughout the body. These leukocytes deliver functioning enzyme to neighboring enzyme-deficient cells via a bystander effect called “cross-correction.” The leukocytes from the modified HSCs continually renew from the engrafted stem cell compartment, ensuring continuous enzyme replacement. This therapy can provide lasting benefits, as demonstrated in Canadian clinical trail (NCT02800070). We aim to expand this effective enzyme correction therapy to broader trail in USA and throughout the world.
