Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression

Invention Grant

US11261447B2 Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression 有权

Patent Title: Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
Application No.: US16630800

Application Date: 2018-07-12
Publication No.: US11261447B2

Publication Date: 2022-03-01
Inventor: David V. Erbe , Tracy L. McGregor
Applicant: Alnylam Pharmaceuticals, Inc.
Applicant Address: US MA Cambridge
Assignee: Alnylam Pharmaceuticals, Inc.
Current Assignee: Alnylam Pharmaceuticals, Inc.
Current Assignee Address: US MA Cambridge
Agency: McCarter & English, LLP
Agent Maria Laccotripe Zacharakis; Deborah L. Nagle
International Application: PCT/US2018/041891 WO 20180712
International Announcement: WO2019/014491 WO 20190117
Main IPC: C12N15/113
IPC: C12N15/113 ; A61P13/12 ; A61K9/00 ; A61K45/06

Abstract:

The invention relates methods of using RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.

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IPC分类:

C	化学；冶金
C12	生物化学；啤酒；烈性酒；果汁酒；醋；微生物学；酶学；突变或遗传工程
C12N	微生物或酶；其组合物；繁殖、保藏或维持微生物；变异或遗传工程；培养基（微生物学的试验介质入C12Q1/00）。
C12N15/00	突变或遗传工程；遗传工程涉及的DNA或RNA，载体（如质粒）或其分离、制备或纯化；所使用的宿主（突变体或遗传工程制备的微生物本身入C12N1/00、C12N5/00、C12N7/00；新的植物入A01H；用组织培养技术再生植物入A01H4/00；新的动物入A01K67/00；含有插入活体细胞的遗传物质以治疗遗传疾病的药剂的应用，基因疗法入A61K48/00，一般肽入C07K）
C12N15/09	.DNA重组技术
C12N15/11	..DNA或RNA片段；其修饰形成（不用于重组技术的DNA或RNA入C07H21/00）
C12N15/113	...调节基因表达的非编码核酸，如反义寡核苷酸