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Saturday, October 10, 2020

A huge disappointment!

The Nobel Prize in Chemistry was just awarded to two scientists for research on CRISPR genetic editing technology. Emmanuelle Charpentier of the Max Planck Unit for the Science of Pathogens and Jennifer Doudna of the University of California Berkeley were named for their work with CRISPR as applied to prokaryotes (bacteria and archaea). If you don't now yet, CRISPR is a method of using a guide RNA and a specific enzyme to "edit" a genome with targeted precise locations in the DNA. While there is still a long way to go to see the clinical use of it, in theory CRISPR can be used to cut out, insert, or replace sections of DNA to correct mutations that cause disease. No need to say, this is one of the most revolutionary technologies in human history!🌹 

While these two brilliant scientists as the very first ones to pilot this revolutionary invention are by all means qualified for this reward and honor, it is a huge disappointment that the Nobel Prize committee did not even mention another important contributor to CRISPR, Dr. Feng Zhang.  While Charpentier and Doudna were focused on CRISPR as applied to bacteria, Zhang's research was focused on using CRISPR as applied to eukaryotes – plants, animals, and humans. As a matter of fact,  nearly all of the research and work in the biotech field on CRISPR is focused on applying the technology to eukaryotes, which is based on Zhang's work. Although not in the context of the Nobel Prize, Zhang will be referred to and named again and again as the practical applications of this wonderful technology become widened and more progressed.  These are advances like the end of genetically caused human disease, crops that are naturally pest- and drought-resistant, and the ability to "grow" organs in animals to then transplant to humans… just to name a few.

In terms of clinical use, we are still at the early stage, largely in phase I studies. So there will be at least a few years before we may see any real use of it in treating human diseases. As with any new technologies, it will not be an easy smooth way in progressing it to real life application. We may see some setbacks along the way but I'm confident it will eventually be successful. 

There are a few CRISPR companies already publicly listed. Two major ones are EDIT and CRSP. As you can see below, both have been largely going sideways since IPO in 2016, likely reflecting the fact that their products are still at the early stage of testing with high risks involved. I'm personally convinced for the efficacy of the technology in treating genetic diseases but the huge unknown or uncertainty is its safety. For example, will the genetic editing cause unwanted alterations of some organ functions? Or will it cause cancers down the road after the gene being manipulated? There is no clear answer to such questions and we just need to wait and see. So be mindful of the potential wide swings of the stocks due to unexpected news from the ongoing studies.  





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