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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.
Exscientia is a global pharmatech company using patient-first artificial intelligence (AI) to discover better drugs, faster. Our mission is to encode, automate and transform every stage of the drug design and development process, by combining the latest AI techniques with experimental innovation, to enable the design of patient centric drug candidates with an improved probability of success. Our validated platform has delivered the first three AI-designed drugs to enter clinical trials and is the first AI system proven to improve clinical outcomes in oncology. We`ve significantly accelerated pre-clinical drug discovery, with 10x productivity improvement in delivering a drug candidate compared with industry standards. By actively applying AI to precision engineer medicines more rapidly and efficiently, we allow the best ideas of science to rapidly become the best medicines for patients.
Repare Therapeutics is developing new, precision oncology drugs for patients that target specific vulnerabilities of tumor cells. Its approach integrates insights from several fields of cell biology including DNA repair and synthetic lethality. Repare`s platform combines a proprietary, high throughput, CRISPR‐enabled gene editing target discovery method with high‐resolution protein crystallography, computational biology and clinical informatics.
We are a biotechnology leader that discovers, develops and delivers innovative therapies to improve the lives of patients with neurodegenerative diseases, hematologic conditions and autoimmune diseases. Patients worldwide benefit every day from our medicines for multiple sclerosis (MS) and hemophilia. Founded in 1978, we are one of the world’s oldest independent biotechnology companies. Our development pipeline is focused on areas of high unmet medical need, as we pursue new therapies for patients suffering from diseases with few or no treatment options. These include programs for secondary progressive MS, Alzheimer’s Disease, spinal muscular atrophy, lupus and amyotrophic lateral sclerosis (ALS), amongst others. With more than 7,500 people worldwide, we are truly a global organization, headquartered in Cambridge, Massachusetts, which is also home to our research operations. Our international headquarters are based in Zug, Switzerland and we have world-class manufacturing facilities in North Carolina and Denmark. We offer therapies globally through direct affiliate presence in 30 countries and a network of distribution partners in over 50 additional countries.
Kowa Research Institute is a Morrisville, NC-based company in the Healthcare, Pharmaceuticals, and Biotech sector.