公开(公告)号：US20250066781A1

公开(公告)日：2025-02-27

申请号：US18782724

申请日：2024-07-24

Applicant: Stoke Therapeutics, Inc.

Inventor： Baruch Ticho ,  Kimberly Parkerson ,  Meena Meena ,  Susovan Mohapatra

IPC: C12N15/113 ,  A61P25/08

Abstract: Provided herein are methods for treating conditions and diseases characterized by SCN1A, SCN8A or SCN5A protein deficiency by targeting the alternative splicing events in SCN1A gene and modulating the expression level of functional proteins in Dravet Syndrome patients and/or inhibit aberrant protein expression.

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：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：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：US20210371866A1

公开(公告)日：2021-12-02

申请号：US17052874

申请日：2019-05-03

Applicant: STOKE THERAPEUTICS, Inc.

Inventor： Isabel AZNAREZ

IPC: C12N15/113 ,  A61K38/46

Abstract: Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient LAL protein expression or a subject having Cholesteryl Ester Storage Disease.

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.