AstraZeneca has reported the first clinical data with its oral PCSK9 inhibitor AZD0780, which it hopes could offer an alternative to current injectable drugs used to lower cholesterol. The new ...
Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment.
CRISPR-Cas9 genome editing exploits the CRISPR-Cas system to modify a genome in a targeted manner. Guided by RNA, the Cas9 endonuclease breaks DNA at a target sequence. Imprecise repair of the ...