It has been a decade since CRISPR/Cas9 gene editing was first proposed in a paper authored by the PhD duo of Jennifer Doudna and Emmanuelle Charpentier. This method effectively “democratized” the gene editing space by providing a workaround for developing custom-engineered gene cutters for specific genome sites, according to Craig Mickanin, global head of the genomic sciences group at Novartis Institutes for Biomedical Research. He believes that the latent trend of accelerated innovation in the genome editing field, as evidenced by the rise of base and prime editing, wouldn’t have been feasible without making CRISPR widely available.
Some of the formative undertakings in the CRISPR-to-therapy pipeline are now coming to fruition. Vertex and CRISPR Therapeutics have announced a campaign to file their own CRISPR/Cas9-edited cell therapy exagamglogene autotemcel (exa-cel) for sickle cell disease and beta thalassemia for rolling review with the FDA. That approval would make it the first market-ready CRISPR-based therapy.
Intellia Therapeutics, fresh from being a pioneer in demonstrating that systemic infusion of CRISPR could help treat disease, also reported promising early-stage data for its in vivo gene editing candidates for transthyretin amyloidosis (ATTR) and hereditary angioedema. The company is positioning a lipid nanoparticle as the ideal delivery mechanism for CRISPR to cripple genes in the liver. While ex vivo gene editing therapies might be more in vogue at the moment, Intellia has turned heads with its in-body direct editing, indicating that the in vivo method may be the future of gene editing.