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Nutcracker Therapeutics is an RNA therapeutics company that has combined the power of advanced engineering with high-precision biosynthesis to deploy a complete RNA therapeutics platform. Armed with this high-tech advantage, we have developed a wholly owned pipeline of RNA therapeutic programs and established partnership initiatives with top clinical investigators at several leading institutions across the globe. With our platform`s ability to accelerate the development of life-changing RNA therapeutics, we seek to advance breakthrough RNA therapies at high velocity through all stages of development across a variety of indications. Our technology platform has the potential to significantly reduce costs and cycle times for RNA therapeutic development, with dramatic advantages in capacity scaling and point-of-care delivery over other RNA manufacturing approaches.
Mirus Bio is a Madison, WI-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.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases.