12/03/09 Marco Island, Florida – The science of RNA interference is moving rapidly toward marketable cures.
Our DNA is, in effect, locked and protected in a cellular clean room without a door. DNA does, however, send out messages with the order to turn genes on or off. Those messages are RNA, or ribonucleic acid.
Therefore, the right RNA sequence can be introduced to the body to modify the behavior of virtually any gene. This is RNA interference (RNAi) and it provides the ability to control specific genes and the proteins these produce. Those proteins, in turn, are the key to most human diseases. The companies that own those therapies will, in turn, become the new pharm giants — or they will be acquired by existing pharma, delivering enormous profits to stockholders.
The challenge now is the delivery of RNAi drugs to cells. RNA is a large fragile molecule that doesn’t penetrate cellular membranes under normal circumstances. Also, the body tends to clear itself of RNAi drugs almost immediately, whether through the kidneys or inside the cell itself. Nuclease, which exists inside the cell, breaks down RNA. For this reason, intense research has been concentrating on carrier molecules that can transport the RNA as a payload.
There are several companies racing to bring the first RNAi therapies to the market. With such a transformational potential in the treatment of human disease, we’ve made it a priority to build a portfolio of the most important RNAi pure plays. Each has a different approach to solving the delivery problem.
As I’ve said before, we can’t know yet which will yield the big solutions. It may be that only one of these companies will break though… We know from a study of past transformational technologies, such as computer chips and software, that the best method of earning transformational profits is to buy a diversified portfolio of the best players and hold them for the long run. That’s what we’re doing in regard to RNAi.
Patrick Cox
Patrick Cox brings two decades of experience in software, public policy, medical economics and biotechnology to the Agora Financial table. He is editor of the Breakthrough Technology Alert and Technology Profits Confidential, as well as a contributor to the Penny Sleuth. Cox, who spent his early childhood on the Crow Indian Reservation in Montana, studied at Boise State University and has written for USA Today, Wall Street Journal, Los Angeles Times and Reason Magazine. Cox has also been featured in The Baltimore Sun and CNBC Business Magazine.
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Let’s see it treat disease. It’s one thing to put an RNAi onto a plate of cultured cells and observe specific gene silencing, another to see a phenotype in an organism. Until that happens, I’m not putting my money in it, because I am not convinced it will ever work. Look how long monoclonals took to take root, and all of the biochemistry is in their favor. You outline in your piece how the biochemistry works against RNAi. It looks like a sucker’s bet.