What is autologous ex vivo gene therapy?

Orchard’s autologous ex vivo gene therapy uses a patient’s own blood stem cells (“autologous”) to correct a genetic disorder. Stem cells are taken from the patient (step 1) and modified outside of the body (“ex vivo”) using a viral vector carrying a functioning copy of the missing or faulty gene (step 2). The gene-modified cells are then transplanted back into the body (step 3).

Watch the video of Bobby Gaspar, Orchard's chief scientific officer describing the technology.

Our pipeline

Orchard is utilising the potential of ex vivo autologous gene therapy to address severe and life-threatening inherited disorders, including primary immune deficiencies, inherited metabolic disorders and blood disorders.

Orchard’s gene therapy pipeline includes Strimvelis®, the first autologous ex vivo gene therapy approved by the EMA in 2016, 3 programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (Wiskott–Aldrich syndrome) and ADA-SCID (adenosine deaminase severe combined immunodeficiency), 2 other clinical programs in X-CGD (X-linked chronic granulomatous disease) and beta-thalassemia, as well as an extensive preclinical pipeline.

Please refer to the section Disease areas and pipeline for more information.

Over 150 patients treated with Orchard’s autologous ex vivo gene therapies in the development phase

Evolved from over 20 years of academic research, Orchard has developed a unique expertise in the manufacturing, preclinical and clinical development of gene therapies for rare diseases. To date, more than 150 patients have been treated with autologous ex vivo gene therapy across five different disease areas, with evidence of sustained clinical effects up to 18 years post treatment in some patients.

What is the scientific advantage of autologous ex vivo gene therapy?

Autologous ex vivo gene therapy is distinct from allogeneic stem cell transplantation, which requires a suitable third-party donor and carries a risk of complications such as graft-versus-host disease. Autologous ex vivo gene therapy uses the patient’s own cells. The donor and the recipient are the same person (hence the term “autologous”) and therefore autologous ex vivo gene therapy provides a perfect cell match. Therefore, there is no risk of graft-versus-host disease or graft rejection. In addition, the modification of the patient's cells outside the body (“ex vivo”) allows us to engineer the patient’s cells to improve clinical outcomes.

Potential benefits of autologous ex vivo gene therapy include:

  • Providing a perfect cell match (the donor and the recipient are the same person)
  • Removing the need to find a suitable third-party donor
  • Eliminating the risk of graft-versus-host disease and graft rejection
  • Leveraging the ability of stem cells to renew and differentiate into daughter cells of multiple lineages
  • Using milder conditioning regimens compared with allogenic stem cell transplantation (in some diseases)
  • Engineering blood stem cells before they are infused back into the patient to achieve a desired clinical effect (for example, over-expressing a gene to improve the uptake in different tissues)
  • A simple, intravenous mode of administration


Our technology explained