公开(公告)号：US20240216369A1

公开(公告)日：2024-07-04

申请号：US17755435

申请日：2020-10-30

Applicant: Novartis AG

Inventor： Martin BEIBEL ,  Beth BOROWSKY ,  Jang-Ho CHA ,  Thomas FALLER ,  Baltazar GOMEZ-MANCILLA ,  Caroline GUBSER KELLER ,  Marc LAISNEY ,  Wen LIN ,  Nicole RENAUD ,  Rajeev SIVASANKRAN

IPC: A61K31/501 ,  A61K9/00 ,  A61K9/08 ,  A61K45/06 ,  A61K47/40 ,  A61P25/28

CPC classification number: A61K31/501 ,  A61K9/0053 ,  A61K9/08 ,  A61K45/06 ,  A61K47/40 ,  A61P25/28

Abstract: Use of a splicing modulator for a treatment slowing progression of Huntington's disease.

公开(公告)号：US20220307053A1

公开(公告)日：2022-09-29

申请号：US17629286

申请日：2020-07-24

Applicant: Novartis AG

Inventor： Martin BEIBEL ,  Caroline Gubser KELLER ,  Dmitriy LUKASHEV ,  Nicole RENAUD ,  Nikita RUDINSKIY ,  Rajeev SIVASANKARAN

IPC: C12N15/86 ,  C12N15/113 ,  C12N15/67 ,  C12N5/079 ,  C12N5/0793

Abstract: Provided herein are compositions comprising minigenes comprising splice modulator binding sequences, for regulatable gene expression, and systems and methods of use thereof.