公开(公告)号：US20220118000A1

公开(公告)日：2022-04-21

申请号：US17379793

申请日：2021-07-19

Applicant: Stoke Therapeutics, Inc. ,  Cold Spring Harbor Laboratory

Inventor： Isabel Aznarez ,  Huw M. Nash ,  Adrian Krainer

IPC: A61K31/7125 ,  A61K47/68 ,  C12N15/113 ,  A61P43/00 ,  A61K38/00 ,  A61K38/17 ,  C12N15/62 ,  C12N15/11

Abstract: Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a disease described herein.

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：US20220088058A1

公开(公告)日：2022-03-24

申请号：US17356147

申请日：2021-06-23

Applicant: Cold Spring Harbor Laboratory ,  Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Huw M. Nash ,  Adrian Krainer

IPC: A61K31/7125 ,  A61K31/712 ,  C12Q1/6883 ,  C12N15/113 ,  C12Q1/68 ,  A61K31/713 ,  A61P43/00

Abstract: Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.

公开(公告)号：US11083745B2

公开(公告)日：2021-08-10

申请号：US16062286

申请日：2016-12-14

Applicant: Cold Spring Harbor Laboratory ,  Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Huw M. Nash ,  Adrian Krainer

IPC: C07H21/04 ,  A61K31/7125 ,  A61K31/712 ,  C12Q1/6883 ,  C12N15/113 ,  C12Q1/68 ,  A61K31/713 ,  A61P43/00

Abstract: Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.

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：US10913947B2

公开(公告)日：2021-02-09

申请号：US16561952

申请日：2019-09-05

Applicant: Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Zhou Han

IPC: C12N15/113 ,  A61P25/12 ,  A61K9/00 ,  A61P25/00

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：US20250059535A1

公开(公告)日：2025-02-20

申请号：US18658390

申请日：2024-05-08

Applicant: Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Kiat Huat Lim ,  Jacob Kach

IPC: C12N15/113

Abstract: Agents that target a processed mRNA, e.g., the 5′ UTR of the processed mRNA, can modulate protein expression, e.g., via modulation of translation of the processed mRNA. Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression. Agents that target the alternative splicing events in genes can modulate the expression level of proteins. Therapeutic agents, which can modulate protein expression by targeting a processed mRNA and/or alternative splicing events, can promote functional protein expression in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease associated with protein deficiency and/or mitochondrial function deficit.

公开(公告)号：US20240254488A1

公开(公告)日：2024-08-01

申请号：US18545753

申请日：2023-12-19

Applicant: Stoke Therapeutics, Inc.

Inventor： Isabel Aznarez ,  Jacob Albert Kach ,  Ana Corrionero Saiz

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/314 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3233

Abstract: Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant or reduced 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 deficiency of the protein.

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.