Market research firm Allied Market Research forecasts that RNA-based therapies will generate $1.2 billion in revenue by 2020, a compounded annual growth rate of 28.4%, according to a recent report. The prediction implies successful commercialization--a long-awaited milestone for the nascent RNA interference (RNAi) treatment paradigm, which shows plenty of promise, but has produced no approved therapies so far, in large part due to drug delivery challenges.
But Allied Market Research points to large-scale public and private funding, the development of novel delivery technologies, and anticipated savings to the healthcare system as drivers of the RNA therapeutics market. It's the gene-silencing potential of RNA therapies that is appealing to pharma and biotech companies, Allied says.
RNAi accomplishes gene silencing by cleaving messenger RNA within cells' cytoplasm, preventing gene transcription from taking place. The naturally occurring paradigm's discovery in 1998 earned scientists Andrew Fire and Craig Mello a Nobel Prize in 2006.
Allied says funding from the U.S. National Institutes of Health and Tekmira's Fast Track designation from the FDA of its RNAi therapeutic for Ebola are also encouraging signs.
In addition to RNAi, RNA antisense is another treatment paradigm in the RNA therapies market, Allied says. Isis Pharmaceuticals ($ISIS) has commercialized two RNA antisense products. They do not face the same delivery challenges as RNAi therapies, which are forecast to garner the most interest in the future.
Allied expects oncology to become the largest market segment for the RNA-based treatments. But The Scientist points out that none of the RNAi candidates have yet made it to Phase III trials. Dicerna's treatment for multiple myeloma and lymphomas is in Phase I and Tekmira recently launched a Phase I/II trial for treating hepatocellular cancer. But the most ambitious company is Israel's Silenseed because it is targeting the notorious oncogene K-RAS, says The Scientist. The company's delivery approach is striking; it aims to inject RNAi encased in a millimeter-scale matrix directly into the tumor.
The liver is the most promising organ for RNAi uptake and treatments because it has 100-nanometer pores that are large enough for lipid nanoparticles containing RNAi to sneak through, says The Scientist. The nanoparticles are necessary to protect the RNAi, which degrades if delivered directly into the bloodstream. Alnylam ($ALNY) is the leader in the liver arena, with 15 RNAi treatments in development, including Phase III candidate Patisiran.
Many companies use lipid nanoparticles to deliver RNAi to the correct site in the cell, but Alnylam attaches the molecules to a sugar molecule, enabling subcutaneous (as opposed to intravenous) delivery. A third delivery strategy is to transport the RNAi in a viral vector, The Scientist explains.
Following Novartis' ($NVS) April decision to shutter its once-ambitious RNAi research efforts and a slump in biotech stocks, confidence in RNAi research was low, leading Alnylam COO Barry Greene to tell FierceDrugDelivery that Big Pharma "has never been able to innovate" and "has been a miserable barometer of high impact technologies." The Allied Market Research forecast should add to the treatment's paradigm's recent momentum. It has already generated a Nobel Prize, and if commercialization occurs, RNAi-drug dollars will be generated as well.