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Zodasiran, an RNAi therapeutic targeting ANGPTL3, for treating patients with homozygous familial hypercholesterolaemia (GATEWAY): an open-label, randomised, phase 2 trial
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DOI：10.1016/S2213-8587(25)00290-6.png)
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Abstract
En 中文
ANGPTL3 plays a key part in lipoprotein metabolism. Zodasiran, a liver-targeted RNA interference therapeutic, inhibits ANGPTL3 expression and reduces atherogenic lipoproteins through mechanisms independent of the LDL receptor (LDLR). This approach is relevant to patients with homozygous familial hypercholesterolaemia (HoFH) who have extreme elevations of LDL cholesterol due to markedly impaired LDLR function and, as a result, very high risk of premature adverse cardiovascular events. We aimed to evaluate the long-term safety and efficacy of zodasiran in patients with HoFH.
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210
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