Status in this atlas: Current approval status is not summarized in this therapy record. A trial phase or a brand name does not establish approval for this use.
Targets ANGPTL3 messenger RNA to reduce hepatic ANGPTL3 protein production.
Uses, doses and availability depend on the formulation, indication and country. Study doses below describe research; they are not prescribing instructions.
The molecule
GalNAc-conjugated siRNA with two 21-nucleotide, chemically modified strands; the sense strand also carries inverted abasic subunits.
Small interfering RNA
Zodasiran
GalNAc-conjugated siRNA with two 21-nucleotide, chemically modified strands; the sense strand also carries inverted abasic subunits.
The original phase 1 paper describes the molecular architecture and publishes its sequence in Supplementary Figure 3. No validated whole-drug 3D coordinates are included.
A next milestone is not summarized for this therapy. This does not mean development has ended.
Development history3 selected milestones
Zodasiran · compound history across indications
Selected sourced milestones, not a complete development history. Dates distinguish study starts, publications, announcements and approvals; the first listed date is not necessarily when development began.
Study start
First phase 1 participant randomized
The primary paper dates the first randomization in the ARO-ANG3 phase 1 basket study to January 4, 2019. The program enrolled healthy participants and cohorts with different lipid disorders.
Nature Medicine reported first human proof of concept for the compound later named zodasiran: suppression of ANGPTL3 with reductions in triglycerides and atherogenic lipoproteins after single and repeated dosing.
The plotted randomized ARCHES-2 trial reported triglyceride and other lipid changes in adults with mixed hyperlipidemia. These biomarker findings do not establish a reduction in cardiovascular events.