What no one is saying about the new CRISPR Gene Therapy

Recently, news broke that Intellia Therapeutics, a pharmaceutical company that focuses on CRISPR gene editing received FDA clearance to use their drug dubbed NTLA-2002 in broader clinical trials. Honestly, that's not really very interesting. This isn't the first gene editing drug tested in humans or even the first CRISPR drug tested in humans. What no one is talking about is the significance that this gene therapy doesn't use viruses!

Almost every gene therapy these days use viruses and currently every FDA approved gene therapy uses viruses to get the gene therapy into cells. I've long been an advocate _against_ viral delivery methods because they are very short-sighted and have many drawbacks. They can usually only hold a small amount of DNA, humans develop resistance to the viral capsid after one dose and they are extremely difficult to produce at scale. Nucleic acids offer a great alternative, they can be unlimited in size, are easy to produce and humans don't develop resistance to them. The problem with nucleic acids is that they are less efficient than viruses at getting into the cell and are not as long lasting.

Using mRNA is a good balance. mRNA doesn't need to go into the nucleus, it can be translated in the cytoplasm so it is more efficient than DNA gene therapies that need to get into the nucleus. If you're using it for CRISPR gene editing it doesn't matter if the mRNA is only in the cell short-term because the gene edit knockout of the KLKB1 gene, as seen on NTLA-2002 would be permanent. This is pretty cool.

We really need to push to move beyond viral gene therapies. In my opinion their use is very short-sighted. So much money are being burned on viral based systems that have so many drawbacks. The fact that a company like Intellia is pushing a non-viral gene therapy is really promising as it will increase the accessibility of gene therapy by lowering the cost and make them easier to produce at scale.