CRISPR genome editing has enormous potential as a means to treat genetic disorders, such as sickle cell disease. However, despite massive success in pre-clinical experiments, the impact of CRISPR in the clinic has been relatively limited.
One problem is a lack of options for large-scale delivery of CRISPR enzymes to patients.
Earlier this month, Aldevron and Synthego co-sponsored a GEN webinar exploring exciting new research into how to deliver genome editing enzymes in vivo into patient cells.
The main presentation was given by Ross Wilson, Ph.D, Assistant Adjunct Professor at the University of California’s Innovative Genomics Institute. His laboratory is working on using ribonucleoproteins (RNPs) as a CRISPR enzyme delivery mechanism. RNPs are the functional complex of CRISPR protein and synthetic guide RNA (sgRNA) that can edit or restore the function of a gene.
RNPs have many advantages over traditional CRISPR enzyme delivery methods, such as viral vectors. The payload enzymes, such as Cas9, only exist within the patient cells for 12-24 hours, which reduces immunological side effects and off-target editing. Moreover, RNPs are made out of recombinant components, which can be scaled up more easily than viral vectors.
In his talk, Dr. Wilson gave an overview of the most exciting pre-clinical work using RNPs to edit cells, including optimising CAR-T production. He also described his own laboratory’s work on adding new functions to RNPs, such as a molecular GPS for targeting specific cell types. Comparing RNPs to a Swiss Army Knife for genome editing, he explained how giving the knife a ‘slippery outer coating’ may improve how easily RNPs distribute through body tissues.
Here at Aldevron, we have pioneered the supply of CRISPR proteins for RNP manufacture. In 2017, we became the first company to offer a GMP Cas9 nuclease as a catalogue product suitable for clinical work. offer the industry’s first GMP-compliant workflow for RNP production.
Through our work, and our partnership with Synthego, who supply research and GMP-quality sgRNA, we’re excited to see how our customers have scaled up their Cas9 requirements. We look forward to how Dr. Wilson and his collaborators will translate their early successes in pre-clinical RNP studies into new, affordable treatments for genetic disease.