A one-time treatment. The potential for lifelong impact.
Orchard is building on decades of research and advancements in the field of gene therapy to develop potential cures for genetic and other severe diseases. Our approach harnesses the unique power of a patient’s own genetically modified blood stem cells, also known as hematopoietic stem cells, or HSCs, to potentially correct the underlying cause of a genetic disease permanently using a single administration.
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” or by extracting bone marrow.
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 of the body, or ex vivo, in a laboratory. The corrected cells are then 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 center, then thawed and infused into the patient intravenously. 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 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 160 people have been treated in clinical trials with our ex vivo autologous gene therapies across six different disease areas, with up to 10 years post-treatment follow-up in some conditions.
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