Orchard Therapeutics is a global fully integrated biopharmaceutical company dedicated to transforming the lives of patients with rare, often-fatal diseases through one-time potentially curative gene therapies. Orchard has demonstrated proof-of-concept of its ex vivo gene therapy approach in five different serious rare diseases, including inherited immune diseases, blood cell disorders and diseases of the brain, which have historically been challenging or nearly impossible to treat. The company expects to file for regulatory approval for its three lead programs by 2021. These include programs in metachromatic leukodystrophy (MLD), severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) and Wiskott-Aldrich syndrome (WAS). Orchard also has three additional clinical programs, as well as an extensive preclinical pipeline. Orchard’s portfolio includes, Strimvelis®, the first ex vivo gene therapy approved by the European Medicines Agency.
Company HQ State:
Company HQ Country:
Main Therapeutic Focus:
Lead Product in Development:
Gene Therapy for MLD and ADA-SCID, two ultra-rare conditions
Development Phase of Primary Product:
Number Of Unlicensed Products (For Which You Are Seeking Partners):