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SalioGen Therapeutics is advancing the Exact DNA Integration Technology (EDIT) platform, the only genome engineering technology that is mammal-derived, for use in potentially curative non-viral gene therapy.
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.
Celladon is a privately held biotechnology company founded with the goal of developing molecular therapies for cardiovascular diseases. Breakthroughs in the basic understanding of the cardiovascular networks combined with powerful new technologies have provided the foundation for new, rational, and technologically sophisticated approaches to cardiovascular treatments. Our first product candidate is designed to target the key enzyme deficiency in advanced heart failure which regulates calcium cycling and contractility in heart muscle cells.
Defyrus Inc. is a Toronto, ON-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Axovant Sciences is a leading clinical-stage neurology company focused on the treatment of dementia and related neurological disorders. We aspire to lead the way into a new era of progress to protect the fullness of life for people living with neurologic conditions. We see the potential of compelling therapeutic pathways and set our sights high on creating life-changing advancement for millions of people affected by neurologic disorders.