The platform is designed to deliver a clinical grade RNP complex generated from a CRISPR nuclease and guide RNA (gRNA)
Aldevron is now offering a GMP ribonucleoprotein (RNP) manufacturing service.
The company developed this platform process for manufacturing RNP complexes using its off-the-shelf GMP grade CRISPR proteins. Aldevron introduced catalog GMP CRISPR proteins in 2017.
Aldevron can use both wild-type SpCas9 and high-fidelity SpyFi Cas9 nucleases for RNP manufacturing, and also can custom manufacture unique nucleases. In addition, the platform includes a robust set of analytical methods to help characterize the RNP final product, including nuclease activity and quantification of bound and unbound components post-RNP complex generation.
“Our clients were searching for a one-reagent solution, and that is what GMP RNPs provide,” said Tom Foti, President of Aldevron’s protein business unit. “We have created unique methods to manufacture and characterize RNP complexes produced from GMP CRISPR nucleases and guide RNAs. Our clients will use these RNPs to help create the future of genetic medicines with the launch of novel cell and gene therapies.”
There are three main CRISPR delivery formats: virus, RNA and RNP. RNPs provide a delivery strategy that circumvents transcription and translation. This makes them immediately active and quickly degraded, which reduces the risk of off-target cutting activity. RNPs are also highly titratable, which allows for precise dosage with controlled and efficient delivery to cells that are difficult to transduce with viruses.