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AveXis, now a Novartis Company, is a clinical-stage gene therapy company relentlessly focused on bringing gene therapy out of the lab and into the clinical setting for patients and families devastated by rare and life-threatening neurological genetic d...
Santarus, Inc. is one of the leading companies in the Healthcare, Pharmaceuticals, & Biotech sector.
Molecular Templates is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of a next-generation of immunotoxins for the treatment of cancers. Our Engineered Toxin Bodies (ETBs) platform represent a new class of targeted biologic therapies that possess unique biological properties.
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.
BionorImmuno is a Bethesda, MD-based company in the Healthcare, Pharmaceuticals, and Biotech sector.