Arrowhead ARO-APOC3 is eligible for FDA grant

FCS is a serious rare genetic disease with a prevalence of 1/1000000, usually caused by a single gene mutation that causes very high levels of triglycerides, usually more than 900 mg/dLrecently, Arrowhead(http:// announcedthat the u.SFood andDrug(http://Administration (
FDA(http://has granted ARO-APOC3 an orphan drug (ODD) for the treatment of family-borne celiac syndrome (FCS)on theof ARO-APOC3ARO-APOC3 is an RNAi therapy that is injected underthels, targeted by lipoprotein C-III (APOC3), and is currently being developed to treat patients with severe hypertriglyceride and FCSIn mid-March this year, the first human study to evaluate ARO-APOC3 (NCT03783377) completed the first batch of subjectsthis study is a Phase I single-dose and multi-dose increment study to assess the safety, tolerance, pharmacokinetics, and pharmacodynamics effects of ARO-APOC3 on adulthealth (http:// volunteers, patients with hyperglycerin trilipidemia, AND PATIENTs with FCS The study planned to recruit 63 subjects 　　At present, ApoC-III has become a highly promising therapeutic target for the reduction of triglycerides ApoC-III regulates lipoprotein (TRL) rich in triglycerides and is a known lipoprotein lipoenzyme (LPL) activity and LPL-mediated TRL fat breakdown inhibitor ApoC-III also delays the liver's removal of lipoprotein residues by inhibiting the receptor-mediated intake of liver cells human genetic studies have shown that people with a functional lysine mutation have a reduced risk of cardiovascular disease and lower plasma triglycerides, but their phenotypes are normal