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miRagen Therapeutics, Inc., is a biopharmaceutical company discovering and developing innovative microRNA (miRNA)-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. From its founding, miRagen has developed ongoing, collaborative relationships with academic institutions that give the company unique access to the most promising early-stage discoveries. This access to groundbreaking science provides a significant strategic advantage in the rapidly-developing field of miRNA-targeting therapeutics.
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.
Tactiva is an immuno-oncology company based in Buffalo, New York, utilizing an innovative, dual T cell receptor (TCR) approach to adoptive T cell therapy.
Insmed is a global biopharmaceutical company focused on the needs of patients with rare diseases. Headquartered in Bridgewater, NJ, Insmed is a dynamic and rapidly growing company with a strong pipeline of drug candidates. At Insmed, we are on a mission to transform the lives of patients battling serious rare diseases. We are developing novel, targeted therapies to help serve the critical unmet needs of these patients. In concert with developing therapeutic solutions, we are committed to improving the entire patient experience. “Insmed is a team of talented and motivated professionals who are dedicated to making a difference in the lives of patients with rare diseases, said Will Lewis, president and CEO at Insmed.
Progenika is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.