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broad range of previously untreatable ills, including certain cancers, rare genetic disorders, and inflammatory diseases. Leading companies in the RNA-based drug development field include Cambridge, MA-based Alnylam Pharmaceuticals (NASDAQ: ALNY) and Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS), which now has 32 antisense-based drugs in its development pipeline.
Antisense technology is based on discoveries in the late 1990s that determined many diseases are caused by abnormal, harmful proteins produced by specific gene mutations. An antisense drug is intended to prevent a mutated gene from producing disease-causing proteins by binding to messenger RNA molecules—before the abnormal protein can be produced.
Alnylam, founded in 2002, has set out to be the leader in RNA interference, another method for blocking the production of disease-related proteins. It has spent the past several years trying to effectively deliver its small interfering RNA molecules (siRNAs) into cells, often using lipid nanoparticles. In late August, Alnylam reported successful early stage animal studies that used advanced lipid nanoparticles to deliver antisense drugs for Transthyretin (TTR)-mediated amyloidosis, an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene.
Aside from moving fast, Arcturus is using some uninhibited superlatives to describe its RNAi technologies, which the company is developing for the treatment of rare diseases.
“We claim to have a superior LNP [liquid nanoparticle] delivery technology than the one [Alnylam] is using in its in Phase 2 [TTR] study,” Chivukula tells me. Arcturus says it has been working with Tekmira Pharmaceuticals (NASDAQ: TKMR) of Vancouver, B.C. in a planned study to deliver Tekmira’s RNAi therapeutic for chronic Hepatitis B infection. Tekmira and Alnylam once worked together as partners, but had a high-profile dispute over intellectual property that culminated in a legal settlement last November. Alnylam agreed to pay Tekmira $65 million to resolve the litigation between the two companies.
Arcturus describes its approach as “best-in-class delivery technology,” and says the technology also enhances the company’s ability to nominate additional clinical candidates throughout 2014 as it expands its own drug pipeline.
“We can also say that we have one of the most potent LNPs [lipid nanoparticles] in the industry right now (we will be presenting this data sometime early next year),” Chivukula writes in an e-mail. In a statement from the company, Arcturus CEO Joseph Payne adds, “We are leading the way in pioneering disruptive advances in this space that will truly impact many people and be a game-changer within the medical field.”
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