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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.
MicroDose Therapeutx is a Monmouth Junction, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Paragon Biosciences invests in and launches biopharmaceutical companies to develop novel therapies for severe medical conditions which do not yet have adequate treatments. Our mission is to help people live longer, healthier lives. Paragon advances that mission through its contribution of Bioscience Innovation Capital(TM): For rare and complex diseases, Paragon analyzes the viability of potential therapies, invests in new biopharmaceutical companies to further develop promising treatments, catalyzes the progress of its portfolio companies through early-incubation services, and provides clinical and regulatory guidance by a team with a near-perfect track record of first-cycle novel drug approvals.
Cyagen Biosciences is a 400-employee company headquartered in Santa Clara, California, with additional locations in Germany, Japan and China. Cyagen is the world`s largest provider of custom-engineered mouse and rat models. Cyagen`s services have become well known for their top quality, full guarantee, and competitive prices.
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare diseases. Our shared purpose to serve patients in need drives us to find answers, change the world, and create a legacy. Alexion`s three highly innovative therapies treat patients with four severe and ultra-rare diseases, and we are committed to developing additional therapies that have the potential to transform patients` lives. Today, Alexion is advancing the most robust rare disease pipeline in the biotech industry, which, in addition to our complement and metabolic clinical programs, includes more than 30 diverse preclinical programs across a range of therapeutic modalities. Alexion has more than 3,000 employees serving patients in 50 countries. Our global headquarters and research operations are based in New Haven, Connecticut and our EMEA headquarters are in Zürich, Switzerland. We also have global supply chain and operations headquarters in Ireland, manufacturing facilities in the United States, and local and regional operations in countries around the world. Alexion was ranked number 5 by Science magazine in its Top Employer Survey and has also been ranked as one of the top companies on the Forbes "World`s Most Innovative Companies" list every year since 2012.