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United States Biological Corporation is a Marblehead, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
Rhythm is a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity.
The vast majority of human disease is governed by the epigenome, the subtle machinery that controls the volume of gene expression in cells. Tune Therapeutics is pioneering the creation of epi-therapeutic medicines with its powerful and precise epigenomic control platform, TEMPO. Unlike genome editing, TEMPO dials the volume of gene expression up or down towards healthy levels, without breaking or permanently rewriting DNA – and it can do this in multiple genes at once. This genetic tuning approach allows us to change cell fate and function at will, unlocking the ability to reverse pathways of cancer, genetic disease and aging. Our approach effectively expands the potential reach of genetic medicine – from targeting a limited range of rare conditions, to overcoming thousands of complex and chronic diseases that currently have no curative treatment.
Isis Pharmaceuticals Inc is a Carlsbad, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.