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Orphan Star Therapeutics, LLC, a biopharmaceutical company, develops therapies for rare genetic diseases including Canavan disease, glut1 deficiency, and rare genetic skin diseases.
Concerro is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Audentes is a biotechnology company committed to the development and commercialization of innovative new medicines for people with serious, rare muscle diseases. We are a passionate, energetic team driven by the goals of improving the lives of patients and building a successful business. We take pride in strong, global relationships with the patient, research, and medical communities.
Galenea Corp is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Regulus is focused on the discovery and development of microRNA therapeutics. microRNAs are members of a large class of non-coding RNAs of approximately 22 nucleotides in length that regulate most genes in the genome. A single microRNA can target and regulate up to hundreds of genes, and these genes are involved in biological networks or pathways. Dysregulated microRNA expression is involved in the initiation of many complex multi-factoral diseases, including cancer, inflammatory disease, fibrosis, and metabolic disease. microRNA therapeutics are oligonucleotide medicines that modulate the function of microRNAs, correcting the imbalance of gene expression and associated cellular pathways to treat human disease. Regulus was formed in September 2007 by Alnylam Pharmaceuticals (NASDAQ:ALNY) and Isis Pharmaceuticals (NASDAQ:ISIS). Regulus benefits substantially from the collective therapeutic RNA expertise of its founding companies, including established proprietary oligonucleotide-based technologies, and broad and dominant intellectual property estates specific to chemically modified oligonucleotides. Regulus has access to over 900 patents and patent applications pertaining to oligonucleotide technologies useful for targeting microRNA therapeutics, and over 170 patents and patent applications directed to microRNA specific technologies. Regulus is located in La Jolla, California and is led by a seasoned executive team and board of directors, with proven expertise in corporate management, business operations, drug discovery and drug development. Regulus’ scientific advisory board consists of world-class scientists and several of the foremost authorities in the field of microRNA research.