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Regeneron NEJM publication on ANGPTL3Genetic and Pharmacologic Inactivation of ANGPTL3 and Cardiovascular Disease http://www.nejm.org/doi/full/10.1056/NEJMoa1612790?query=featured_home CONCLUSIONS: In conclusion, we observed that loss-of-function variants in ANGPTL3 were associated with a reduced risk of coronary artery disease, mirroring the antiatherogenic effects of inhibition of Angptl3 by monoclonal antibody in mice. In human volunteers, monoclonal antibody inhibition of ANGPTL3 was associated with dose-dependent reductions in LDL cholesterol and triglyceride levels. Genetic and therapeutic antagonism of ANGPTL3 in humans and of Angptl3 in mice was associated with decreased levels of all three major lipid fractions and decreased odds of atherosclerotic cardiovascular disease. (Funded by Regeneron Pharmaceuticals and others; ClinicalTrials.gov number, NCT01749878.) Relevance to Alnylam: ALNY are developing ALN-ANG, an RNAi therapeutic targeting the gene angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia. Maybe, like with PCSK9, the monoclonals can pave the way, and we can come in with a (potentially!) 'better mousetrap'?? |
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