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WO2021247995A3 - Compositions and methods of treating neuropathic pain - Google Patents

Compositions and methods of treating neuropathic pain Download PDF

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Publication number
WO2021247995A3
WO2021247995A3 PCT/US2021/035898 US2021035898W WO2021247995A3 WO 2021247995 A3 WO2021247995 A3 WO 2021247995A3 US 2021035898 W US2021035898 W US 2021035898W WO 2021247995 A3 WO2021247995 A3 WO 2021247995A3
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WO
WIPO (PCT)
Prior art keywords
methods
neuropathic pain
treating neuropathic
compositions
sirna
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.)
Ceased
Application number
PCT/US2021/035898
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French (fr)
Other versions
WO2021247995A2 (en
WO2021247995A8 (en
Inventor
Dinah Wen-Yee Sah
Steven Paul
Jinzhao Hou
Pengcheng ZHOU
Qingmin Chen
Markus Hossbach
Jochen DECKERT
Wen Jin
Cansu COLPAN
Jeffrey Brown
Hatim ZARIWALA
Matt KELLEY
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.)
Voyager Therapeutics Inc
Original Assignee
Voyager Therapeutics Inc
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Filing date
Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of WO2021247995A2 publication Critical patent/WO2021247995A2/en
Publication of WO2021247995A3 publication Critical patent/WO2021247995A3/en
Publication of WO2021247995A8 publication Critical patent/WO2021247995A8/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • 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
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The present disclosure relates to small interfering RNA (siRNA) molecules against the mutant, variant, and/or wild type SCN3A, SCN9A, SCN10A and/or SCN11A gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating neuropathic pain using the siRNA molecules and AAV vectors.
PCT/US2021/035898 2020-06-04 2021-06-04 Compositions and methods of treating neuropathic pain Ceased WO2021247995A2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202063034557P 2020-06-04 2020-06-04
US63/034,557 2020-06-04
US202163140580P 2021-01-22 2021-01-22
US63/140,580 2021-01-22
US202163185418P 2021-05-07 2021-05-07
US63/185,418 2021-05-07

Publications (3)

Publication Number Publication Date
WO2021247995A2 WO2021247995A2 (en) 2021-12-09
WO2021247995A3 true WO2021247995A3 (en) 2022-01-13
WO2021247995A8 WO2021247995A8 (en) 2022-02-03

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2021/035898 Ceased WO2021247995A2 (en) 2020-06-04 2021-06-04 Compositions and methods of treating neuropathic pain

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WO (1) WO2021247995A2 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024169770A1 (en) * 2023-02-17 2024-08-22 苏州时安生物技术有限公司 Sirna inhibiting scn9a gene expression, pharmaceutical composition and use thereof
WO2024249240A2 (en) * 2023-05-26 2024-12-05 Eli Lilly And Company Novel scn10a rnai agents and uses thereof
TW202521696A (en) * 2023-11-20 2025-06-01 大陸商上海舶望製藥有限公司 Compositions and methods for inhibiting expression of sodium voltage-gated channel alpha subunit 9 (scn9a)
WO2025128589A1 (en) * 2023-12-13 2025-06-19 Eli Lilly And Company Compositions and methods for modulating nav1.8
WO2025140551A1 (en) * 2023-12-29 2025-07-03 Neuro3 Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING NaV1.7 (SCN9A) -ASSOCIATED DISORDERS

Citations (5)

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Publication number Priority date Publication date Assignee Title
WO2009033027A2 (en) * 2007-09-05 2009-03-12 Medtronic, Inc. Suppression of scn9a gene expression and/or function for the treatment of pain
WO2018007980A1 (en) * 2016-07-06 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of pain related disorders
WO2018051175A1 (en) * 2016-09-16 2018-03-22 Olipass Corporation Scn9a antisense oligonucleotides
WO2018138585A1 (en) * 2017-01-24 2018-08-02 Olipass Corporation Scn9a antisense pain killer
WO2018165504A1 (en) * 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing

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