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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.
Enzon Pharmaceuticals Inc. is one of the leading companies in the Healthcare, Pharmaceuticals, and Biotech sector.
MediciNova, Inc., a development stage biopharmaceutical company, focuses on acquiring and developing small molecule therapeutics for the treatment of serious diseases for the United States market. The company’s principal product candidates include MN-166, an ibudilast-based drug candidate, which is in Phase I and Phase II clinical trials for the treatment of drug dependence and pain; and MN-221, a ß2 -adrenergic receptor agonist that has completed Phase II clinical trials for the treatment of acute exacerbations of asthma. It also has other product candidates in the clinical development for the treatment of acute exacerbations of asthma, multiple sclerosis and other central nervous system disorders, bronchial asthma, interstitial cystitis, solid tumor cancers, generalized anxiety disorders/insomnia, preterm labor, urinary incontinence, and thrombotic disorders. MediciNova, Inc. was founded in 2000 and is headquartered in La Jolla, California.
CepTor Corporation is a Cockeysville, MD-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Ambys Medicines is a biotechnology company focused on discovering and developing regenerative and restorative therapies for people with advanced liver disease. Founded by world-renowned experts in liver disease and regenerative medicine, Ambys is pioneering the application of novel modalities including cell and gene therapy, and gain-of-function drug therapy, to meet the urgent need for treatments that have the potential to restore liver function and prevent the progression to liver failure across multiple liver diseases that are untreatable or poorly treated today. Ambys was launched in 2018 by Third Rock Ventures and Takeda Pharmaceuticals with $140M in starting capital and is headquartered in South San Francisco, Calif.