Orchard is building on decades of research and advancements in the field of gene therapy to develop potential cures 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.
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The patient’s own (“autologous”) blood stem cells, also called hematopoietic stem cells (HSCs), are harvested 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 (into a vein). Once the cells engraft in the bone marrow, they begin to self-renew and produce healthy 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.
Our approach will potentially make a profound difference in the lives of those affected by certain rare, inherited diseases. To date, more than 150 people have been treated in clinical trials with our ex vivo autologous gene therapies across six different disease areas, with up to 18 years post-treatment follow-up in some conditions.