Ionis获FDA批准开发降低甘油三酯的罕见病药物

IF 33.1 1区 生物学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
{"title":"Ionis获FDA批准开发降低甘油三酯的罕见病药物","authors":"","doi":"10.1038/s41587-024-02546-z","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"43 1","pages":"5-5"},"PeriodicalIF":33.1000,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Ionis wins FDA nod for triglyceride-lowering rare disease drug\",\"authors\":\"\",\"doi\":\"10.1038/s41587-024-02546-z\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":19084,\"journal\":{\"name\":\"Nature biotechnology\",\"volume\":\"43 1\",\"pages\":\"5-5\"},\"PeriodicalIF\":33.1000,\"publicationDate\":\"2025-01-17\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Nature biotechnology\",\"FirstCategoryId\":\"5\",\"ListUrlMain\":\"https://www.nature.com/articles/s41587-024-02546-z\",\"RegionNum\":1,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"BIOTECHNOLOGY & APPLIED MICROBIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Nature biotechnology","FirstCategoryId":"5","ListUrlMain":"https://www.nature.com/articles/s41587-024-02546-z","RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
引用次数: 0

摘要

美国食品和药物管理局已批准Ionis制药公司的药物Tryngolza (olezarsen)作为低脂饮食的辅助药物,用于治疗家族性乳糜小铁血症综合征(FCS)。这种以rna为靶点的药物是首个被批准用于治疗这类罕见遗传疾病的药物。患有这种疾病的人体内无法分解乳糜微粒(由大约90%的甘油三酯组成的脂蛋白颗粒)。在美国,这种疾病影响了大约3000人。FCS导致血浆中空腹甘油三酯危险的高水平(500 mg dl-1),因为遗传上缺乏分解脂质的能力,导致急性胰腺炎的风险增加。有针对性地减少载脂蛋白C-3 (APOC3),一种在肝脏中产生的蛋白质,是甘油三酯代谢的关键角色,可以纠正这种生化紊乱。Tryngolza是一种针对肝脏APOC3 mRNA的n -乙酰半乳糖胺偶联反义寡核苷酸。通过抑制APOC3蛋白的产生,这种疗法提高了人体分解脂肪的能力,降低了循环中富含甘油三酯的脂蛋白,提高了动脉粥样硬化的风险。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Ionis wins FDA nod for triglyceride-lowering rare disease drug
求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
Nature biotechnology
Nature biotechnology 工程技术-生物工程与应用微生物
CiteScore
63.00
自引率
1.70%
发文量
382
审稿时长
3 months
期刊介绍: Nature Biotechnology is a monthly journal that focuses on the science and business of biotechnology. It covers a wide range of topics including technology/methodology advancements in the biological, biomedical, agricultural, and environmental sciences. The journal also explores the commercial, political, ethical, legal, and societal aspects of this research. The journal serves researchers by providing peer-reviewed research papers in the field of biotechnology. It also serves the business community by delivering news about research developments. This approach ensures that both the scientific and business communities are well-informed and able to stay up-to-date on the latest advancements and opportunities in the field. Some key areas of interest in which the journal actively seeks research papers include molecular engineering of nucleic acids and proteins, molecular therapy, large-scale biology, computational biology, regenerative medicine, imaging technology, analytical biotechnology, applied immunology, food and agricultural biotechnology, and environmental biotechnology. In summary, Nature Biotechnology is a comprehensive journal that covers both the scientific and business aspects of biotechnology. It strives to provide researchers with valuable research papers and news while also delivering important scientific advancements to the business community.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信