RNAi and RNA companies had a terrible time when they were abandoned by big pharmaceutical companies. The abandonment came from the fact that both types of biotechs lacked proper delivery methods to get the RNA molecule strands to their target. Well it seems that this year all that has changed completely, as RNA therapeutics have been creating delivery methods to make their technologies work. Some RNA technologies use nanoparticles, lipids, and other forms of delivery. I will be discussing one RNAi company, and another RNA company that long term investors should put on their radars for some nice gains.
The RNAi to consider
The first is an RNAi biotech named Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY). Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) has run up a lot from 2012 to 2013, edging up 195%! In the same time period, the Dow has gone up 84%! Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is an RNAi therapeutic that’s a bit different than the others. The reason being is Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is targeting orphan diseases, which carry lot more benefits than non orphan diseases. Some advantages include longer market exclusivity, longer patents, and faster approval by the FDA.
Just recently, Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) reported results of its trial targeting a rare disease known as TTR-Amyloidosis. This disease causes a lot of abnormal mutations of proteins that accumulate in the liver and other organs. This condition is very severe because of the organs that are involved. Why investors should look into this is that orphan drugs get the whole market because of limited competition. Also, they can set high prices as there is no other therapy available for these patients. The results showed a knockdown of genes by 93%, which for a phase 2 trial is very good. Investors should watch closely as other trials are reported. The company is also targeting other rare diseases like Hemophilia and Hypercholesterolemia.
Now to RNA
The next company deals with RNA molecules, and this company is known as ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS). This company is amazing in its own right, as it has a big pipeline of antisense drugs. It uses a different method of delivery as it binds its drugs with the RNA molecule itself, and not the proteins. The company goes after diseases with unmet medical needs like Hypercholesterolemia and other rare diseases.
This company has partnerships with Biogen Idec Inc (NASDAQ:BIIB) and GlaxoSmithKline plc (ADR) (NYSE:GSK) in the rare diseases department. The drug that ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) has developed for hypercholesterolemia is Kynamro, which has been approved by the FDA on Jan. 29, 2013. ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) is expected to eventually make up to $2 billion on the Kynamro drug. The fact is that Sanofi will get 50% of the profit of Kynamro sales. Still, this first antisense drug to be approved by the FDA shows that the ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) technology works in terms of efficacy. With its big pipeline and ground breaking technology, I think ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) will be a good long-term play.