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We are a genome editing company developing genetic medicines for the fight against rare pediatric diseases. GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV based gene therapies With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types.
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.
Kadmon is a fully integrated biopharmaceutical company engaged in the discovery, development and commercialization of small molecules and biologics to address disease areas of significant unmet medical need. We are developing product candidates within autoimmune and fibrotic diseases, oncology and genetic diseases. We leverage our multi-disciplinary research and clinical development team members, who prior to joining Kadmon had brought more than 15 drugs to market, to identify and pursue a diverse portfolio of novel product candidates, both through in-licensing products and employing our small molecule and biologics platforms. We utilize our advanced understanding of the molecular mechanisms of disease to establish development paths for disease areas where significant unmet medical needs exist.
Volastra aims to extend the lives of patients with cancer by leveraging unique insights into chromosomal instability. Moving away from primary tumor models to target the site of metastasis in the toughest-to-treat solid tumors, we are altering the treatment paradigm for metastatic cancers.
Pars Environmental is a Trenton, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.