CRISPR Therapeutics Investor Relations Material

CRISPR-Cas9 had its initial description ten years ago when a study revealed the potential of exploiting the system for RNA-programmable gene editing. It allows scientists to change the molecules that carry the instructions to produce proteins without changing the original DNA code.

The technology has its foundation in the natural defense mechanism of bacteria and single-celled microorganisms. When mentioned, CRISPR often links to Cas9, the protein that helps target and cut the genetic code to be edited. You can therefore see Cas9 as the genetic scissors cutting the DNA at a specific point, and once the gene is cut, there are two possible outcomes:

1. The cell repairs itself.

2. The researchers insert a new DNA template for the repair.

CRISPR technology is transforming medicine, enabling us to treat and prevent many diseases. It also has the potential to be used to make precise changes, such as replacing faulty genes – true gene editing.

We have seen remarkable progress within this technology in the first ten years. As new editing technologies develop, we can insert larger DNA fragments more precisely, enabling us to tackle more of the 75,000 genetic variants associated with human disease. Some of CRISPR’s main competitors include Intellia Therapeutics, Denali Therapeutics, Vaxcyte, and Ginkgo Bioworks.