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Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology`s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic.
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.
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