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Acureon Pharmaceuticals is a Berwyn, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
ProtoKinetix, Inc. is a molecular biotechnology company that has developed and patented a family of hyper stable, potent glycopeptides (AAGP™) that dramatically enhance the therapeutic results and reduce the cost of stem cell medicine. Due to the anti-inflammatory effect of AAGP™ molecules, the Company is currently targeting the direct treatment of diseases that have a major inflammatory component.
Code Bio was founded on the belief that more can be done for people living with debilitating genetic diseases. We are driven by the potential of our transformative technology to treat the untreatable and cure the incurable. Code Bio is developing highly targeted genetic medicines with its proprietary, novel, multivalent, synthetic DNA delivery platform, 3DNA®, which has been engineered to overcome many of the challenges inherent with the delivery of genetic medicines. 3DNA offers unparalleled tissue and cell targeting specificity, improved bioavailability, capability to deliver large genetic payloads, potential ability to re-dose, and a scalable, modular, reproducible manufacturing process. Our 3DNA delivery platform is poised to transform the field of genetic medicines, enabling targeted delivery of gene therapy, RNAi and other genetic modalities. Currently we are advancing an internal pipeline of genetic medicines focused on select diseases without a cure (Duchenne`s Muscular Dystrophy and Type 1 Diabetes), as well as establishing partnerships to take forward programs in both rare and prevalent diseases.
Celator Pharmaceuticals is a Princeton, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.