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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.
Quellis was founded to deliver best-in-class therapies to patients suffering serious rare diseases – and underserved by current treatment options. The Company is based in Boston, and is led by partners from the biotech incubator Viridian LLC in collaboration with team at biotech accelerator Xontogeny LLC, and with funding from the Perceptive Xontogeny Venture Fund. The Quellis team has deep experience in mAb discovery and development, company creation, and private and public biotech investment. Our shared goal is to create meaningful medicines for every disease we target.
Aspen is developing induced-pluripotent stem cells (iPSCs) to address diseases with high unmet medical need, beginning with autologous neuron replacement for both sporadic and genetic forms of Parkinson`s disease (PD) and extending across the brain and affected organs.
Enthrive is a New York, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
REVOLUTION Medicines discovers and develops new drugs that harness the therapeutic potential of frontier oncology targets on behalf of cancer patients. Our innovative product engine draws inspiration from nature and evolution, which offer insights into cancer targets, disease mechanisms and therapeutic approaches. We pay particular attention to natural products that are inherently rich with biological function as a result of natural selection and can inform and guide our discovery and development of small molecules that can modulate these targets.