Orchard is building on decades of research and advancements in the field of gene therapy to develop potential treatments for rare and often-fatal diseases. Our ex vivo autologous gene therapy approach is designed to use a person’s own blood stem cells and insert into those cells a working copy of the missing or faulty gene. By giving these gene-corrected cells to the patient, we aim to permanently correct genetic disorders with a single treatment.
This approach relies on the intrinsic ability of blood stem cells, also known as hematopoietic stem cells or HSCs, to self-renew in a patient’s bone marrow and produce new blood cells of all types. Our approach also avoids the need for an allogeneic stem cell transplantation, which uses cells from a donor and can result in serious complications including graft-versus-host disease.
Learn more about our gene therapy approach:
The patient’s own (“autologous”) blood stem cells, also called hematopoietic stem cells (HSCs), are harvested either through a peripheral blood procedure known as “leukapheresis”.
Once outside the body, special blood-forming HSCs are selected. These are the cells we want to correct as they can develop into many different cell types in the blood.
We use a particular type of virus, a lentivirus that has been disabled from multiplying, to insert a working copy of the missing or faulty gene into the cells. This process happens outside the body, or "ex vivo", in a laboratory. The corrected cells are frozen, or "cryopreserved".
The patient undergoes a conditioning regimen that clears the defective hematopoietic stem cells from the bone marrow and makes space for the genetically corrected cells to engraft.
The gene-corrected cells are transported to a treatment centre, then thawed and infused into the patient intravenously. Once the cells engraft in the bone marrow, they begin to self-renew and produce corrected blood cells of all types. Some of these cells are also able to cross the blood-brain barrier and potentially achieve a therapeutic effect in the brain.
IE-NoP-2300002, January 2024
