Researchers at the University of California in San Diego have developed a way to disguise the genetic material itself so that it can enter cells and shut down genetic diseases at the core.
Alnylam today announced the addition of RNA interference therapies for the hepatitis delta virus and chronic liver infections to its clinical pipeline at the American Association for the Study of Liver Diseases in Boston. The company also said it remains on track to select a candidate for its hepatitis B program by the end of the year and hopes to file an investigational new drug application with the FDA in late 2015.
RNA interference specialist Arrowhead presented new data from an ongoing Phase IIa study of its hepatitis B treatment ARC-520, demonstrating a reduction of the disease's surface antigens in what the company says is a first for RNAi.
RNAi specialist Alnylam is focusing on rare diseases at the moment (with the exception of hepatitis B), but Chief Business Officer Laurence Reid told investors at the Leerink Rare Disease Roundtable conference that the company is also looking to enter bigger markets via partnerships.
RNAi leader Alnylam, the American Porphyria Consortium and The European Porphyria Network are launching the Explore observational study of patients with the rare disease, with the hope of advancing Alnylam's candidate for porphyria, with a goal of filing for approval in late 2014 or early 2015.
Tekmira, developer of an RNAi treatment for Ebola, has come to terms with regulators on an access program for its in-development drug, allowing the company to provide doses to patients who have contracted the deadly virus.
Market research firm Allied Market Research forecasts that RNA-based therapies will generate $1.2 billion in revenue by 2020, a compounded annual growth rate of 28.4%, according to a recent report.
A while ago, there was talk of a dreaded biotech bubble, and Novartis' decision to slam the breaks on its RNA interference research was making waves. But lately, the talk has turned positive on the promising yet unproven treatment paradigm, which utilizes RNAi to deliver gene-based therapy.
Arrowhead Research reported on Aug. 12 that patients in its Phase II clinical trial of the ARC-520 RNA interference candidate for hepatitis B had a "similar" knockdown to those reported in primate studies. The stock price fell at the very end of the trading day to close at $12.00 from an opening-day price of $13.00.
Alnylam announced July 22 that its intellectual property estate has been strengthened due to the U.S. Patent Office's issuance of a Notice of Allowance for claims in one of its patent applications that covers "chemically modified RNA therapeutics conjugated with an N-acetylgalactosamine (GalNAc) ligand independent of length, sequence, or disease target," according to a statement.