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 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 a bone marrow transplant, which uses cells from a donor and can result in serious complications including graft-versus-host disease.
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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.
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