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Clinical trials and meta-analyses demonstrate that ANGPTL3 inhibitors, including SHR-1918 and zodasiran, provide superior LDL-C reduction compared to PCSK9 inhibitors in HoFH, particularly in patients with null-receptor genotypes where trad
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
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 t