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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.
Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and devastating metabolic diseases with limited treatment options. Aeglea`s lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designation. In the second quarter of 2020, the Company initiated a Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria. AGLE-177 has also been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have big impact on the lives of patients and their families.
Western Oncolytics is developing a new generation of oncolytic viral immunotherapies to help patients accross the globe beat cancer
Entrada Therapeutics is a biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines, Endosomal Escape Vehicles (EEV™), to engage intracellular targets that have long been considered inaccessible and undruggable. The Company`s EEV therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues with an improved therapeutic index. Through its proprietary, highly versatile and modular EEV Platform, Entrada is building a robust development portfolio of oligonucleotide-, antibody- and enzyme-based programs for the potential treatment of neuromuscular disease, immunology, oncology and diseases of the central nervous system.
CRISPR Therapeutics is a leading gene-editing company focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. Our multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Our lead programs in beta-thalassemia and sickle cell disease have advanced to IND/CTA-enabling studies with a CTA filing planned by the end of 2017, and we are advancing additional programs in ex vivo and in vivo disease areas. In addition to our fully-owned programs, our strategic collaborations with Bayer AG and Vertex Pharmaceuticals expand our portfolio and enable us with unique capabilities. Through our private financings, partnerships, and IPO we have raised >$400M to fund and accelerate our portfolio. We have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. Our company is headquartered in Zug, Switzerland with R&D operations in Cambridge, Massachusetts, USA and some business operations in London, United Kingdom.