A single injection every 3 months of a novel drug that targets the lipid metabolism enzyme ANGPTL3 resulted in substantial reductions in LDL cholesterol in patients with HoFH in the phase 2 ...

Based on ANGPTL3 knockdown, CRISPR Tx concluded the two lowest doses were minimally active. In the three patients who received the third dose, CRISPR Tx reported mean reductions in triglycerides ...

Mean reductions in ANGPTL3 of 82.7% and 80.1%, respectively ANPTL3 inhibition with ARO-ANG3 also reduced HDL-C, non-HDL-C, and triglycerides, consistent with published human genetic data ...

WASHINGTON — Treatment with vupanorsen, a novel antisense oligonucleotide targeting hepatic ANGPTL3, achieved modest reductions in non-HDL at 24 weeks at all doses studied, according to results ...

“Genetic studies show that ANGPTL3 loss-of-function variants lead to enhanced lipoprotein lipase and endothelial lipase activity, resulting in lower concentrations of most plasma lipoproteins ...

If researchers can knock out the Angptl3 gene, they can let the lipases do their work and reduce levels of cholesterol in the blood. It turns out that some lucky people have a natural mutation in ...

The study, Targeting ANGPTL3 with an antisense oligonucleotide in adults with dyslipidemia (TRANSLATE-TIMI 70), will evaluate various doses of vupanorsen to inform potential future development.

The team then gave experimental mice a single injection of the 306-O12B LNP, packed with mRNA coding for CRISPR-Cas9 and a single-guide RNA targeting Angptl3. The treatment resulted in a profound ...

Targets primarily include PCSK9, ANGPTL3, HSD17B13, KHK, ASGR1, GPR75, CIDEB, and INHBE. Drug types primarily include small molecules, monoclonal antibodies, RNAi, and CRISPR gene editing therapies.

For the PCSK9 and ANGPTL3 product candidates, Lilly now holds the product rights previously held by Beam, including the right to opt-in to share 33% of worldwide development expenses and to ...