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 ex vivo autologous gene therapy approved by the EMA in 2016, three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), 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