Compositions and methods of treating facioscapulohumeral muscular dystrophy

Invention Grant

US12104156B2 Compositions and methods of treating facioscapulohumeral muscular dystrophy 有权

Patent Title: Compositions and methods of treating facioscapulohumeral muscular dystrophy
Application No.: US18052899

Application Date: 2022-11-04
Publication No.: US12104156B2

Publication Date: 2024-10-01
Inventor: Barbora Malecova , Rob Burke , David Sala Cano , Beatrice Diana Darimont
Applicant: Avidity Biosciences, Inc.
Applicant Address: US CA San Diego
Assignee: AVIDITY BIOSCIENCES, INC.
Current Assignee: AVIDITY BIOSCIENCES, INC.
Current Assignee Address: US CA San Diego
Agency: WILSON SONSINI GOODRICH & ROSATI
Main IPC: C12N15/113
IPC: C12N15/113 ; A61K31/7125 ; A61K47/68 ; A61K48/00 ; A61P21/00 ; A61P21/06 ; C07K16/28 ; C07K19/00

Abstract:

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.

Public/Granted literature

Information query

Espacenet

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