RNAi Targeting of ANGPTL3 with Zodasiran Delivers Substantial LDL-C Lowering in Patients with Homozygous Familial Hypercholesterolaemia
Posted inCardiology Diabetes & Endocrinology news

RNAi Targeting of ANGPTL3 with Zodasiran Delivers Substantial LDL-C Lowering in Patients with Homozygous Familial Hypercholesterolaemia

The Phase 2 GATEWAY trial demonstrates that zodasiran, an RNAi therapeutic targeting ANGPTL3, significantly reduces LDL cholesterol in patients with homozygous familial hypercholesterolaemia (HoFH) by approximately 36-40%. The treatment was well-tolerated and showed sustained efficacy, offering a promising LDLR-independent therapeutic avenue for this high-risk population.
First-in-Human CRISPR-Cas9 Editing of ANGPTL3 Shows Dose‑Dependent Protein Knockdown with Acceptable Short‑Term Safety in Phase 1 Trial
Posted inCardiology news

First-in-Human CRISPR-Cas9 Editing of ANGPTL3 Shows Dose‑Dependent Protein Knockdown with Acceptable Short‑Term Safety in Phase 1 Trial

A phase 1, ascending‑dose study of CTX310 (LNP-delivered CRISPR-Cas9 targeting ANGPTL3) in 15 patients produced dose-dependent ANGPTL3 reductions at ≥0.6 mg/kg with few acute safety signals; longer follow‑up and larger trials are required to define efficacy, durability, and long-term risks.