The Next Generation Incubator at CNSI

Orchard Therapeutics is a clinical-stage biotechnology company dedicated to bringing transformative gene therapies to patients with serious and life-threatening orphan diseases.


Orchard is targeting serious and life-threatening disorders with no treatment option (such as Mucopolysaccharidosis IIIA, also known as Sanfilippo syndrome type A or MPS-IIIA) or where existing options leave significant management gaps (such as adenosine deaminase deficiency severe combined immunodeficiency also called ADA-SCID). We believe that addressing such a profound medical need with the transformative efficacy and safety of ex-vivo gene therapy will create a sustainable business model.


Ex-vivo autologous gene therapy uses the patient's own stem cells (“autologous”) to treat an underlying genetic condition. The key steps in the procedure include: taking stem cells from the patient’s bone marrow or peripheral blood; inserting a functioning copy of the missing or faulty gene into the patient’s stem cells outside of the patient’s body (“ex-vivo”); and transplanting the genetically-corrected stem cells back into the patient, after the patient has been given a conditioning regimen to “make room” for the genetically-corrected cells.


  • Lentiviral vectors used with autologous ex-vivo gene therapy are integrating vectors
  • Greater control over genetic manipulation
  • Ability to select cell type to be transduced with viral vector
  • Not associated with immunogenicity

Development Stage:

Pre-clinical and Clinical Orchard’s clinical development pipeline includes novel treatments for primary immune deficiency disorders and inherited metabolic disorders, including ADA-SCID as a lead program and MPS-IIIA as well as other undisclosed late and early stage development programs in other indications.

Select Publications:

Company Contact:

Website: LinkedIn: Contact: Sylvie Blanchier-Franklin,, +44 (0) 20 3823 2149