Feng Zhang, one of the cofounders of Editas Medicine (EDIT, Financial), is at it again with yet another CRISPR discovery. This time it’s a new CRISPR system called CRISPR-cas13d, for a new private company he’s involved in called Arbor Biotechnologies.
Before we break this one down, those interested in CRISPR stocks should know that while developments are happening fast, none are anywhere near marketable drugs, and it could be years before anything even enters the clinic. CRISPR companies are going to have to thrive on investor optimism and new influxes of equity investments for the next several years at least. Valuations could principally depend on the availability of and ease of attracting capital from new sources. Further, since the science is so new and revolutionary and nobody knows exactly where it will lead, pinning a number on a reasonable valuation for these companies is especially difficult.
That said, the science is still very exciting and worth exploring nonetheless.
Arbor is in the business of rapid drug discovery, using a host of state-of-the-art tools including artificial intelligence and gene sequencing among others to catalogue proteins and the genes underpinning them, and then index their different functions and possible therapeutic applications. Compare this to the current standards for discovering monoclonal antibodies for example, which are much slower and arduous, and there is no contest.
The current widely used methods for discovering new antibodies starts with implanting genes into a phage, gathering the antibodies the phage then displays on its cell membrane, testing them on different antigens in vitro until you get the one reactive to a target antigen, and only then testing on mice and so on. Arbor’s system for discovering proteins and their possible therapeutic applications is orders of magnitude faster because it is much more direct. Arbor believes, according to its recent press release, that it will soon be able to simply look at genetic sequencing data to determine protein function and hence predict therapeutic applications directly, just by looking at the code underpinning the proteins.
Using this platform, Arbor discovered CRISPR-cas13d, an enzyme used by bacteria against RNA viruses for defense. The enzyme can cut RNA at specific points. The significance of this is that RNA in humans only translates existing genes from DNA. It is not part of the genome itself, so edited RNA and a patient’s genome remains unaltered, but the temporary instructions change.
What uses could cas13d have? Many well-known genetic diseases are believed to come down to RNA translation errors, including cystic fibrosis, Duchenne muscular dystrophy and Parkinson’s. Not in the sense that errors in the DNA genetic code are irrelevant to these conditions, but in the sense that genetic diseases like these may be able to be corrected by correcting the mistake that RNA picks up from the bad DNA before the RNA translates it into bad protein. In this way, treatment could theoretically be administered without altering a patient’s genome at the root, which could for obvious reasons be dangerous.
We are now at the discovery phase of this industry, and given that the same handful of scientists are involved, like Zhang, the chances of significant roll-up as the industry matures is, I believe, quite high. It may not be imminent per se, but it is a high-probability eventuality.
Disclosure: No positions.
