Some patients with very high LDL-C who have already suffered a heart attack:
*may need both the PCSK9 and the ANGTPL3 pathways inactivated
*can we accomplish this with gene editing -
the switching off of two genes in the liver in the same individual?
#ACC22 @VerveTx $VERV
NHPs treated:
*day 0 with VERVE-101 targeting PCSK9 (one-time IV infusion)
*day 30 with ANGPTL3 base editor (one-time IV infusion)
*necropsy at day 90 -->
high level editing of each gene!!
69% editing PCSK9
61% editing ANGTPL3
Each edit designed to switch off respective gene
Day 0 - VERVE 101 Rx,
blood PCSK9 level plummets
Day 30 - ANGPTL3 base editor Rx,
blood ANGPTL3 level plummets
This will likely be durable for lifetime animal (person)!
With sequential dosing of our two products which are mRNA/guide RNA packaged in an lipid nanoparticle,
no sustained impact on LFTs
this is one of the key advantages of non-viral (LNP) delivery!!
Sequential dosing: no impact on total bilirubin
@VerveTx is advancing
a pipeline of single-course in vivo liver gene editing programs
to safely and durably lower LDL-C
and
treat atherosclerotic cardiovascular disease
#ACC22
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