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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.
MedXcel is a Tampa, FL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Established in 2008, Cardialen, Inc., set out to develop an implantable defibrillator using low-electrical energy to restore normal heart rhythm and potentially avoid the negative side-effects of current therapies.
Sengenics is a functional proteomics company that leverages its patented KREX technology to discover autoantibody biomarker signatures for prediction of drug response and severe immune-related adverse events (irAEs). KREX™ was invented and patented by Professor Jonathan Blackburn whilst he was a member of the faculty at the University of Cambridge. Jonathan is the CSO of Sengenics. The key application of KREX™ is the discovery of autoantibody biomarker signatures for prediction of drug response and severe immune-related adverse events (irAEs). KREX™ can also be used to identify autoantibody biomarkers that may be used to diagnose cancer, autoimmune or neurodegenerative conditions years before conventional diagnostic tests. Some autoantibodies that are identified as diagnostic biomarkers may be protective and have potential in themselves as therapeutic biomolecules.
ElevateBio is accelerating the future of biotechnology. We have a launched a revolutionary new model that integrates innovators, infrastructure and capital to effectively develop cell, gene and related therapies for patients with severe and life-threatening disorders.