Orchard Therapeutics is targeting disorders with no treatment option or where existing options leave significant management gaps. Orchard’s autologous ex vivo gene therapy uses a patient’s own blood stem cells 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).

In April 2018, Orchard Therapeutics raised Series C financing and acquired GSK's rare disease portfolio through a strategic agreement. This acquisition strengthens Orchard's position as a global leader in gene therapy for rare diseases. Read more about it here!