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1. WO2021247995 - COMPOSITIONS AND METHODS OF TREATING NEUROPATHIC PAIN

Publication Number WO/2021/247995
Publication Date 09.12.2021
International Application No. PCT/US2021/035898
International Filing Date 04.06.2021
IPC
C12N 15/113 2010.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/7088 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
CPC
A61K 31/7088
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-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
C12N 2310/14
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2310Structure or type of the nucleic acid
10Type of nucleic acid
14interfering N.A.
C12N 2320/34
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2320Applications; Uses
30Special therapeutic applications
34Allele or polymorphism specific uses
Applicants
  • VOYAGER THERAPEUTICS, INC. [US]/[US]
Inventors
  • SAH, Dinah, Wen-Yee
  • PAUL, Steven
  • HOU, Jinzhao
  • ZHOU, Pengcheng
  • CHEN, Qingmin
  • HOSSBACH, Markus
  • DECKERT, Jochen
  • JIN, Wen
  • COLPAN, Cansu
  • BROWN, Jeffrey
  • ZARIWALA, Hatim
  • KELLEY, Matt
Agents
  • LU, Yu
  • BAI, Mei
  • BAYNE, Christopher, D.
  • ALBURGER, James, M.
  • SUNDIATA, Coatlton, Bennett
Priority Data
63/034,55704.06.2020US
63/140,58022.01.2021US
63/185,41807.05.2021US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS OF TREATING NEUROPATHIC PAIN
(FR) COMPOSITIONS ET MÉTHODES DE TRAITEMENT DE LA DOULEUR NEUROPATHIQUE
Abstract
(EN) 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.
(FR) La présente invention concerne de petite molécules d'ARN interférent (ARNsi) dirigées contre le gène mutant, variant et/ou sauvage de SCN3A, SCN9A, SCN10A et/ou SCN11A, un vecteur de virus adéno-associé (AAV) codant pour des molécules d'ARNsi et des procédés de traitement de la douleur neuropathique à l'aide des molécules d'ARNsi et des vecteurs AAV.
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