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Exonics Therapeutics was launched in February 2017 to advance the research of our scientific founder, Dr. Eric Olson and his laboratory at the University of Texas Southwestern Medical Center (UTSW) to develop treatments for patients with neuromuscular diseases. Dr. Olson is one of the world’s leading experts in the study of muscle cells and the application of gene editing to treat these types of diseases. In particular, Dr. Olson’s laboratory has used adeno-associated virus (AAV) to deliver a CRISPR/Cas9 technology that can identify and repair exon mutations to restore the production of dystrophin, a protein that helps stabilize and protect muscle fibers. Dystrophin is the protein missing in boys with Duchenne. The loss of dystrophin causes Duchenne.
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.
Molecular Therapeutics is a Ann Arbor, MI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Aldevron provides contract manufacturing and scientific services. We specialize in plasmid DNA, protein production, and antibody generation. PLASMID DNA PRODUCTION With the successful completion of over 50,000 projects, we have developed unsurpassed expertise in plasmid manufacturing. Using proprietary technology, Aldevron manufactures DNA for a wide range of research, preclinical, clinical, and diagnostic applications (1 mg to 100 g). PROTEIN PRODUCTION Aldevron offers a wide range of protein services including host cell optimization, expression screening, process development, contract production, enzyme and antibody assay design, and more. Proteins produced by Aldevron are used for a wide range of agriculture and biomedical applications from research grade to full cGMP. ANTIBODY GENERATION GENOVAC Antibody Technology supports all project types from reagent antibodies to diagnostic and therapeutic applications with high success rates. Based on many years of experience, our immunization technology has been developed to cater for all protein targets where other methods have failed (including e.g. GPCRs). Our custom antibodies have been validated in over 130 publications and patents for more than 500 clients worldwide. Additional services include antibody processing, production, labeling, characterization and sequencing.
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.