WO2024036143A3 - Methods for modulating rna splicing - Google Patents

Methods for modulating rna splicing Download PDF

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Publication number
WO2024036143A3
WO2024036143A3 PCT/US2023/071833 US2023071833W WO2024036143A3 WO 2024036143 A3 WO2024036143 A3 WO 2024036143A3 US 2023071833 W US2023071833 W US 2023071833W WO 2024036143 A3 WO2024036143 A3 WO 2024036143A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
rna splicing
modulating rna
modulating
rems
Prior art date
Application number
PCT/US2023/071833
Other languages
French (fr)
Other versions
WO2024036143A2 (en
Inventor
Wencheng Li
Original Assignee
Ptc Therapeutics, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ptc Therapeutics, Inc. filed Critical Ptc Therapeutics, Inc.
Publication of WO2024036143A2 publication Critical patent/WO2024036143A2/en
Publication of WO2024036143A3 publication Critical patent/WO2024036143A3/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4365—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system having sulfur as a ring hetero atom, e.g. ticlopidine

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

A method for modulating an amount or type of a mature RNA transcript, comprising contacting a compound of Formula (I) with a pre-mRNA transcript transcribed from a gene, wherein the gene comprises a DNA REMS sequence and the pre-mRNA transcript transcribed from the gene comprises a RNA REMS sequence.
PCT/US2023/071833 2022-08-09 2023-08-08 Methods for modulating rna splicing WO2024036143A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263370902P 2022-08-09 2022-08-09
US63/370,902 2022-08-09

Publications (2)

Publication Number Publication Date
WO2024036143A2 WO2024036143A2 (en) 2024-02-15
WO2024036143A3 true WO2024036143A3 (en) 2024-03-28

Family

ID=89852473

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/071833 WO2024036143A2 (en) 2022-08-09 2023-08-08 Methods for modulating rna splicing

Country Status (1)

Country Link
WO (1) WO2024036143A2 (en)

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007121326A2 (en) * 2006-04-12 2007-10-25 Crosslink Genetics Corporation Compositions and methods for modulating gene expression
US20170306335A1 (en) * 2014-12-23 2017-10-26 The Broad Institute Inc. Rna-targeting system
US20190330615A1 (en) * 2016-11-28 2019-10-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
WO2020163406A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
US20220135586A1 (en) * 2019-02-13 2022-05-05 Ptc Therapeutics, Inc. Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007121326A2 (en) * 2006-04-12 2007-10-25 Crosslink Genetics Corporation Compositions and methods for modulating gene expression
US20170306335A1 (en) * 2014-12-23 2017-10-26 The Broad Institute Inc. Rna-targeting system
US20190330615A1 (en) * 2016-11-28 2019-10-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
WO2020163406A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
US20220135586A1 (en) * 2019-02-13 2022-05-05 Ptc Therapeutics, Inc. Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia

Also Published As

Publication number Publication date
WO2024036143A2 (en) 2024-02-15

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