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Dicerna Pharmaceuticals is a pharmaceutical company developing RNA interference (RNAi)-based therapeutics against genetically-defined targets in multiple disease areas, including cancer. Dicerna's therapeutic approach utilizes its Dicer Substrate siRNA (DsiRNA) molecules and EnCore drug delivery technologies to silence previously undruggable disease targets, offering a potential new treatment option for patients. Dicerna delivers its DsiRNA molecules to cancer cells using its EnCore lipid nanoparticle drug delivery technology. EnCore has been specifically engineered to accumulate in tumors and mediate efficient delivery of the DsiRNAs to the RNAi machinery in the cancer cell cytoplasm. Encore particles are well tolerated and manufactured using well-established unit operations to ensure commercial scalability.
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing.
Akcea Therapeutics (NASDAQ: AKCA) is a late-stage biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders.
Regulus Therapeutics (NASDAQ: RGLS), a biopharmaceutical company, focuses on the discovery and development of drugs that target RNAs to treat a range of diseases in the United States. The Company uses its microRNA product platform to develop anti-miRs, which are chemically modified and single-stranded oligonucleotides. Its clinical development products include RG-101, a GalNAc-conjugated anti-miR targeting miR-122 to treat patients with hepatitis C virus infection; RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome; and RG-125, a GalNAc-conjugated anti-miR targeting microRNA-103/107 to treat nonalcoholic steatohepatitis in patients with type 2 diabetes/pre-diabetes. The company has strategic alliance with GSK to discover and develop microRNA therapeutics for immuno-inflammatory diseases; Sanofi to discover and develop microRNA therapeutics for fibrotic diseases; AstraZeneca AB to discover and develop microRNA therapeutics for cardiovascular diseases, metabolic diseases, and oncology; and Biogen to evaluate the use of microRNA signatures as a biomarker for human patients with various sclerosis.
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates.
Alnylam Pharmaceuticals (NASDAQ: ALNY) is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company believes that are many new therapeutic frontiers for its RNA technology, including applications in vaccine development, stem cell biology, bio-processing, genomics and biologics.
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