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
Application number
PCT/US2021/035898
Other languages
French (fr)
Other versions
WO2021247995A8 (en
WO2021247995A2 (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
Original Assignee
Voyager 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.)
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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

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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 N.A.
    • 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

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 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

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PCT/US2021/035898 WO2021247995A2 (en) 2020-06-04 2021-06-04 Compositions and methods of treating neuropathic pain

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