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WO2025137637A3 - Wobble base pairs for improving specificity of rna editing - Google Patents

Wobble base pairs for improving specificity of rna editing

Info

Publication number
WO2025137637A3
WO2025137637A3 PCT/US2024/061569 US2024061569W WO2025137637A3 WO 2025137637 A3 WO2025137637 A3 WO 2025137637A3 US 2024061569 W US2024061569 W US 2024061569W WO 2025137637 A3 WO2025137637 A3 WO 2025137637A3
Authority
WO
WIPO (PCT)
Prior art keywords
base pairs
rna editing
wobble base
improving specificity
specificity
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
PCT/US2024/061569
Other languages
French (fr)
Other versions
WO2025137637A2 (en
Inventor
Yiannis SAVVA
Brian Booth
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Shape Therapeutics Inc
Original Assignee
Shape 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 Shape Therapeutics Inc filed Critical Shape Therapeutics Inc
Publication of WO2025137637A2 publication Critical patent/WO2025137637A2/en
Publication of WO2025137637A3 publication Critical patent/WO2025137637A3/en
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Disclosed herein are engineered guide RNAs and compositions comprising the same for treatment of diseases or conditions in a subject. Also disclosed herein are methods of treating diseases or conditions in a subject by administering engineered guide RNAs or pharmaceutical compositions described herein.
PCT/US2024/061569 2023-12-21 2024-12-20 Wobble base pairs for improving specificity of rna editing Pending WO2025137637A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363613139P 2023-12-21 2023-12-21
US63/613,139 2023-12-21

Publications (2)

Publication Number Publication Date
WO2025137637A2 WO2025137637A2 (en) 2025-06-26
WO2025137637A3 true WO2025137637A3 (en) 2025-07-31

Family

ID=96138935

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/061569 Pending WO2025137637A2 (en) 2023-12-21 2024-12-20 Wobble base pairs for improving specificity of rna editing

Country Status (1)

Country Link
WO (1) WO2025137637A2 (en)

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8592643B2 (en) * 2008-01-29 2013-11-26 Central Institute For Experimental Animals Methods for introducing a human gene into a marmoset embryo for making a transgenic marmoset
WO2016180726A1 (en) * 2015-05-08 2016-11-17 Fundació Institut D'investigació En Ciències De La Salut Germans Trias I Pujol Method for in vitro diagnosis of dementia with lewy bodies using alphasynuclein gene transcripts
WO2022103839A1 (en) * 2020-11-11 2022-05-19 Shape Therapeutics Inc. Rna editing compositions and uses thereof
US20230044119A1 (en) * 2019-12-02 2023-02-09 Shape Therapeutics Inc. Therapeutic Editing
WO2023086902A1 (en) * 2021-11-10 2023-05-19 Shape Therapeutics, Inc. Machine-learning based design of engineered guide systems for adenosine deaminase acting on rna editing
US20230174977A1 (en) * 2020-05-26 2023-06-08 Shape Therapeutics Inc. Engineered circular polynucleotides

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8592643B2 (en) * 2008-01-29 2013-11-26 Central Institute For Experimental Animals Methods for introducing a human gene into a marmoset embryo for making a transgenic marmoset
WO2016180726A1 (en) * 2015-05-08 2016-11-17 Fundació Institut D'investigació En Ciències De La Salut Germans Trias I Pujol Method for in vitro diagnosis of dementia with lewy bodies using alphasynuclein gene transcripts
US20230044119A1 (en) * 2019-12-02 2023-02-09 Shape Therapeutics Inc. Therapeutic Editing
US20230174977A1 (en) * 2020-05-26 2023-06-08 Shape Therapeutics Inc. Engineered circular polynucleotides
WO2022103839A1 (en) * 2020-11-11 2022-05-19 Shape Therapeutics Inc. Rna editing compositions and uses thereof
WO2023086902A1 (en) * 2021-11-10 2023-05-19 Shape Therapeutics, Inc. Machine-learning based design of engineered guide systems for adenosine deaminase acting on rna editing

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ZHAO: "Imperfect guide-RNA (igRNA) enables CRISPR single-base editing with ABE and CBE", NUCLEIC ACIDS RESEARCH, 29 March 2022 (2022-03-29), pages 4161 - 4170, XP093028668, DOI: 10.1093/nar/gkac201 *

Also Published As

Publication number Publication date
WO2025137637A2 (en) 2025-06-26

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