Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.
Orchard’s portfolio of autologous ex vivo gene therapies includes Strimvelis, the first autologous ex vivo gene therapy approved by the European Medicines Agency for adenosine deaminase severe combined immunodeficiency (ADA-SCID). Additional programs for primary immune deficiencies, neurometabolic disorders and hemoglobinopathies include three advanced registrational studies for ADA-SCID, metachromatic leukodystrophy (MLD) and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion dependent beta-thalassemia (TDBT) and Mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline.
Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.
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.
|Type||Public / Private|
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Top 5 Recent News Headlines
Orchard Therapeutics Reports Second Quarter 2019 Financial Results and Builds Momentum Ahead of Regulatory Filings
Metachromatic Leukodystrophy (MLD) MAA Submission and Initiation of ADA-SCID Rolling BLA Expected in the First Half of 2020; OTL-103 Recently Granted RMAT Designation for Wiskott-Aldrich Syndrome (WAS) MLD and Mucopolysaccharidosis Type I (MPS-I) Abstracts Accepted for Oral Presentations at SSIEM