Ex-vivo autologous gene therapy:

Our technology

Ex-vivo autologous gene therapy technology makes use of the patient's own stem cells (autologous)

These stem cells are taken from the patient (step 1) and genetically corrected outside of the body (ex-vivo) with a lentiviral vector carrying a functioning copy of the missing or faulty gene (step 2)

The genetically corrected cells are then transplanted back into the body (step 3)

Ex-vivo autologous gene therapy:

The scientific advantage

The use of the patient's own cells (autologous) provides a perfect match. This eliminates the requirement for a donor search and the risk of graft-versus-host disease and transplant rejection, which are major complications of transplants from allogeneic donors (that is, bone marrow, peripheral blood or cord blood transplants from a third party donor)

Also, the correction of the patient's cells outside the body ("ex-vivo") allows us to engineer the cells to improve the therapeutic outcome

Use of patient's own cells ("autologous")
  • No requirement for a donor search
  • Reduced transplant morbidity compared to allogeneic transplants:
    • No risk of graft-versus-host disease / graft rejection
    • Milder conditioning regimen
Correction of patient's cells outside the body ("ex-vivo")
  • Each patient is treated with a unique (personalized) product
  • Ex-vivo autologous gene therapy allows to increase the dose of correct gene given to the patient
  • Ex-vivo autologous gene therapy allows to deliver functional enzyme to the central nervous system in some conditions