公开(公告)号：US11814622B2

公开(公告)日：2023-11-14

申请号：US18304878

申请日：2023-04-21

Applicant: Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Aditya Venkatesh ,  Gene Liau

IPC: C12N15/113 ,  A61P27/02 ,  A61K9/00 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61K9/0048 ,  A61P27/02 ,  C12N15/86 ,  C12N2310/11 ,  C12N2310/20 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2750/14143

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 and/or mitochondrial function deficit.

公开(公告)号：US20230116704A1

公开(公告)日：2023-04-13

申请号：US17832182

申请日：2022-06-03

Applicant: Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Zhou Han ,  Anne Christiansen ,  Meena Meena ,  Baruch Ticho ,  Gene Liau

IPC: C12N15/113 ,  A61P25/08

Abstract: Alternative splicing events in SCN1A gene 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 SCN1A gene can modulate the expression level of functional proteins in Dravet Syndrome patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition caused by SCN1A, SCN8A or SCN5A protein deficiency.

公开(公告)号：US20240102011A1

公开(公告)日：2024-03-28

申请号：US18483333

申请日：2023-10-09

Applicant: Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Aditya Venkatesh ,  Gene Liau

IPC: C12N15/113 ,  A61K9/00 ,  A61P27/02 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61K9/0048 ,  A61P27/02 ,  C12N15/86 ,  C12N2310/11 ,  C12N2310/20 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2750/14143

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 and/or mitochondrial function deficit.

公开(公告)号：US20230250429A1

公开(公告)日：2023-08-10

申请号：US18304878

申请日：2023-04-21

Applicant: Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Aditya Venkatesh ,  Gene Liau

IPC: C12N15/113 ,  C12N15/86 ,  A61P27/02 ,  A61K9/00

CPC classification number: C12N15/113 ,  C12N15/86 ,  A61P27/02 ,  A61K9/0048 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/314 ,  C12N2310/321 ,  C12N2310/20 ,  C12N2750/14143

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 and/or mitochondrial function deficit.

公开(公告)号：US20230287410A1

公开(公告)日：2023-09-14

申请号：US17924966

申请日：2021-04-30

Applicant: Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Aditya Venkatesh ,  Gene Liau

IPC: C12N15/113 ,  A61P27/02

CPC classification number: C12N15/113 ,  A61P27/02 ,  C12N2310/11 ,  C12N2310/314 ,  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 and/or mitochondrial function deficit.