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.

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.

WAVE Life Sciences is utilizing its synthetic chemistry drug development platform to design, develop and commercialize stereopure nucleic acid therapeutics that precisely target the underlying cause of rare genetic diseases, delivering new treatment options for patients. Given the versatility of its chemistry platform, WAVE's pipeline is designed to span multiple oligonucleotide modalities including antisense, exon-skipping and single-stranded RNAi.

Moderna Therapeutics (NASDAQ: MRNA) is a biotechnology company that is focused on drug discovery and drug development based on messenger RNA. It creates synthetic mRNA that can be injected into patients to help them create their own therapies. The company was founded in 2010 and is based in Cambridge, Massachusetts.

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.