CRISPR gene editing is a technique used in molecular biology to modify the genomes of living organisms. It is based on a simplified version of the bacterial CRISPR-Cas9 antiviral defense system. By delivering the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location. This allows existing genes to be removed and/or new ones added in vivo.
In more technical terms, Cas9 is a dual RNA-guided DNA endonuclease enzyme associated with the CRISPR adaptive immune system in Streptococcus pyogenes. The CRISPR-Cas9 genome editing technique was a significant contributor to the Nobel Prize in Chemistry in 2020, which was awarded to Emmanuelle Charpentier and Jennifer Doudna.
The first drug based on CRISPR/Cas9 gene editing, exagamglogene autotemcel (exa-cel), was approved in November 2023 with the trade name Casgevy. It is used in the United Kingdom to cure sickle-cell disease and beta thalassemia. On December 8, 2023, Casgevy was approved in the United States by the FDA. Casgevy is a first-of-its-kind treatment targeting the BCL11A gene, and was discovered by Vertex Pharmaceuticals and CRISPR Therapeutics.
Casgevy is created by enriching autologous cells for CD34+ cells, and then genome editing ex vivo by introducing the CRISPR/Cas9 ribonucleoprotein (RNP) complex by electroporation. The gRNA included in the RNP complex enables CRISPR/Cas9 to make a precise DNA double-strand break at a critical transcription factor binding site (GATA1) in the erythroid-specific enhancer region of the BCL11A gene.
Apart from RNP, DNA or mRNA encoding Cas9 and gRNA is also being investigated in clinical trials.
Yaohai Bio-Pharma Offers One-Stop CDMO Solution for Cas9 Nuclease
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