公开(公告)号：US11896651B2

公开(公告)日：2024-02-13

申请号：US15574420

申请日：2016-04-15

Applicant: Genzyme Corporation

Inventor： Guoxiang Ruan ,  Abraham Scaria

IPC: A61K38/46 ,  A61K31/7105 ,  A61K31/7088 ,  A61P27/02 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90

CPC classification number: A61K38/465 ,  A61K31/7088 ,  A61K31/7105 ,  A61P27/02 ,  C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  C12N2310/20 ,  C12N2750/14143 ,  C12N2800/80 ,  A61K31/7105 ,  A61K2300/00 ,  A61K31/7088 ,  A61K2300/00 ,  A61K38/465 ,  A61K2300/00

Abstract: Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.

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.