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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. 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. Orchard is a publicly traded company (NASDAQ: ORTX) with offices in the UK and the US, including London, San Francisco and Boston.
Bioinformatics.org is a Framingham, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Founded in 2019, Totus Medicines uses revolutionary chemical biology to create life-changing therapies to treat previously untreatable diseases across the entire human genome. Totus is based in Cambridge, Massachusetts.
Dewpoint Therapeutics is leveraging a transformative shift in the understanding of cellular biology regulation to discover and advance breakthrough small-molecule therapeutics.
Pearl Therapeutics is a Redwood City, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.