公开(公告)号：US20240033378A1

公开(公告)日：2024-02-01

申请号：US18299956

申请日：2023-04-13

Applicant: STOKE THERAPEUTICS, INC.

Inventor： ISABEL AZNAREZ ,  Enxuan JING ,  Jacob KACH ,  Aditya VENKATESH ,  Juergen SCHARNER ,  Baruch TICHO ,  Gene LIAU

IPC: A61K48/00 ,  C12N15/113

CPC classification number: A61K48/005 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321

Abstract: Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency.

公开(公告)号：US20210268667A1

公开(公告)日：2021-09-02

申请号：US16758776

申请日：2018-10-23

Applicant: STOKE THERAPEUTICS, INC.

Inventor： Isabel AZNAREZ ,  Enxuan JING ,  Jacob KACH ,  Aditya VENKATESH ,  Juergen SCHARNER ,  Baruch TICHO ,  Gene LIAU

IPC: B25J15/00

Abstract: Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency.