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Translate Bio is a leading mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. Our proprietary mRNA therapeutic platform (MRTTM) is designed to develop product candidates that deliver mRNA carrying instructions to produce intracellular, transmembrane and secreted proteins for therapeutic benefit. We believe that our MRTTM platform is applicable to a broad range of diseases caused by insufficient protein production or where production of proteins can modify disease, including diseases that affect the lung, liver, eye, central nervous system, lymphatic system and circulatory system. Our two lead programs are being developed as treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency.
Perlegen Sciences, Inc. is a Newark, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
CombiMatrix Corporation is a Irvine, CA-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.
Prescos is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.