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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.
Exicure is developing a new class of immunomodulatory and gene silencing drugs against validated targets. Our 3-dimensional, spherical nucleic acid (SNA™) architecture unlocks the potential of nucleic acid therapeutics in multiple organs. Our lead programs address diseases from inflammatory disorders to oncology. SNA constructs overcome one of the most difficult obstacles to nucleic acid therapeutics: safe and effective delivery into cells and tissues. SNA constructs exhibit unparalleled transfection efficiency into numerous cell and tissue types including the skin without carriers or transfection agents. Moreover, SNAs can be used as potent immunotherapeutic agents for the treatment of cancer or infectious disease.
Axogen is the leading company focused specifically on the science, development and commercialization of technologies for peripheral nerve regeneration and repair. We are passionate about helping to restore peripheral nerve function and quality of life to patients with physical damage or discontinuity to peripheral nerves by providing innovative, clinically proven and economically effective repair solutions for surgeons and health care providers. Every day, people suffer traumatic injuries or undergo surgical procedures that impact the function of their peripheral nerves. Physical damage to a peripheral nerve, or the inability to properly reconnect peripheral nerves, can result in the loss of muscle or organ function, the loss of sensory feeling, or the initiation of pain. Axogen`s comprehensive portfolio of products focuses on helping patients touch, run, feel, kiss, smile and hug—some of the many things healthy peripheral nerves allow us to do.
Columbus Children's Research Institute is a Columbus, OH-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Goldfinch Bio is a biotechnology company that is singularly focused on discovering and developing precision therapies for kidney disease. Just as the goldfinch was a prominent figure of the Renaissance, Goldfinch Bio is leading a new age of therapeutic discovery to transform the treatment paradigm for patients with kidney disease. Goldfinch is integrating breakthroughs in kidney genetics and biology to identify new therapeutic targets and advance first-in-class drug candidates to treat patients with kidney disease. Our Product Engine will industrialize the integration of genetics and kidney biology and confer a differentiated ability to identify, validate, and pursue novel therapeutic targets to treat progressive kidney disease. At Goldfinch, we`re pioneering a new approach to discover and develop therapeutics that target the molecular basis of kidney disease. We`re looking for teammates who relish the challenge, and are compelled to leave a positive mark on the lives of colleagues and patients alike.