Orchard's 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 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.

Find out more about the diseases we are targeting with our programs.

Information for patients and caregivers

Information for medical professionals