Join the club for FREE to access the whole archive and other member benefits.

Orchard Therapeutics

Company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies

Orchard Therapeutics is a leading global fully integrated commercial-stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies.

Orchard’s portfolio of autologous ex vivo gene therapy programs has demonstrated sustained clinical benefit in over 150 patients across five disease areas. Theseprograms include 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.

The company is partnered with world-leading institutions in gene therapy, including University College London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University Hospitals, the University of California Los Angeles and Boston Children’s Hospital, and Telethon Institute of Gene Therapy/Ospedale San Raffaele.

Visit website: https://www.orchard-tx.com/

 orchard-therapeutics

 orchard_tx

Details last updated 03-Oct-2019

Orchard Therapeutics News

NHS offers the world's most expensive drug for children with a rare disease

BBC - 04-Feb-2022

Libmeldy is life-saving gene therapy now available for use in England

Read more...
Topics mentioned on this page:
Gene Therapy