Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 | NEJM Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein cholest...

www.nejm.org/doi/full/10....
#CTX310 targets #ANGPLT3 protein to edit the gene with one infusion to lower #LDL & #triglycerides.
This is a very small, early trial incorporating #CRISPR techniques.

$CRSP out with #CTX310 #ANGPTL3 CRISPR in hypercholesterolemia/mixed dyslipidemia.

Highest dose (n=1) w/ substantial efficacy...but 0.8mg/kg 'lean body weight-based dose' sounds rather high for an LNP. Imp to learn more about liver tox, esp that n=1 subject.

www.globenewswire.com/news-release...