Orchard Therapeutics

About:

Orchard Therapeutics is a biotechnology company dedicated to assisting patients through innovative gene therapies.

Website: https://www.orchard-tx.com

Twitter/X: orchard_tx

Top Investors: Temasek Holdings, RA Capital Management, F-Prime Capital, Pavilion Capital, Cormorant Asset Management

Description:

Orchard Therapeutics is a fully integrated commercial-stage company dedicated to treating 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. These programs 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.