公开(公告)号：US20240384271A1

公开(公告)日：2024-11-21

申请号：US18626784

申请日：2024-04-04

Applicant: Genzyme Corporation

Inventor： Seng Cheng ,  Sarah Melissa Jacobo ,  Takako Moriguchi ,  Catherine O'Riordan ,  Guoxiang Ruan

IPC: C12N15/113 ,  A61K45/06 ,  A61P21/00 ,  C12N15/86

Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat DM1.

公开(公告)号：US20230365968A1

公开(公告)日：2023-11-16

申请号：US18296283

申请日：2023-04-05

Applicant: Genzyme Corporation

Inventor： Seng Cheng ,  Sarah Melissa Jacobo ,  Takako Moriguchi ,  Catherine O'Riordan ,  Guoxiang Ruan

IPC: C12N15/113 ,  C12N15/86 ,  A61P21/00

CPC classification number: C12N15/113 ,  C12N15/86 ,  A61P21/00 ,  C12N2750/14143

Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat DM1.