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1. WO2019131829 - COMPOSITION FOR MODIFYING TARGET GENE

Publication Number WO/2019/131829
Publication Date 04.07.2019
International Application No. PCT/JP2018/048034
International Filing Date 27.12.2018
IPC
C12N 15/09 2006.1
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
A01K 67/027 2006.1
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
67Rearing or breeding animals, not otherwise provided for; New breeds of animals
027New breeds of vertebrates
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
A61K 38/46 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
A61K 47/18 2017.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
16containing nitrogen
18Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
A61K 47/24 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
24containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
CPC
A01K 2217/00
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2217Genetically modified animals
A01K 2227/10
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2227Animals characterised by species
10Mammal
A01K 67/027
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
67Rearing or breeding animals, not otherwise provided for; New breeds of animals
027New breeds of vertebrates
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
A61K 31/7105
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
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 38/46
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
Applicants
  • 国立大学法人京都大学 KYOTO UNIVERSITY [JP]/[JP]
  • 武田薬品工業株式会社 TAKEDA PHARMACEUTICAL COMPANY LIMITED [JP]/[JP]
Inventors
  • 堀田 秋津 HOTTA Akitsu
  • 井福 正隆 IFUKU Masataka
  • 藤本 直子 FUJIMOTO Naoko
  • 岩渕 久美子 IWABUCHI Kumiko
  • 見城 江利也 KENJO Eriya
  • 蒔田 幸正 MAKITA Yukimasa
  • 落合 留美子 OCHIAI Rumiko
Agents
  • 大野 聖二 OHNO Seiji
  • 梅田 慎介 UMEDA Shinsuke
  • 今野 智介 IMANO Tomoyuki
Priority Data
2017-25479828.12.2017JP
Publication Language Japanese (ja)
Filing Language Japanese (JA)
Designated States
Title
(EN) COMPOSITION FOR MODIFYING TARGET GENE
(FR) COMPOSITION DE MODIFICATION DE GÈNE CIBLE
(JA) 標的遺伝子改変用組成物
Abstract
(EN) A delivery method is provided which delivers a gene modifying tool which achieves a high gene modification efficiency in cells. This composition, for inducing genetic modification in a target locus in a cell, contains 1) the compound represented by expression (I) or a salt thereof, 2) structural lipids, and 3) guide RNA or DNA containing a sequence encoding this, and/or RNA-guided nuclease, or nucleic acids including a sequence encoding this. In expression (I), n is an integer 2-5, R is a linear C1-5 alkyl group, a linear C7-11 alkenyl group or a linear C11 alkadienyl group, the wavy lines independently represent cis or trans bonds.
(FR) La présente invention concerne un procédé d'administration fournissant un outil de modification de gène qui atteint une efficacité élevée de modification de gène dans les cellules. Cette composition, pour induire la modification génétique dans un lieu cible dans une cellule, contient 1) le composé représenté par l'expression (I) ou un sel de ce dernier, 2) des lipides structurels, et 3) de l'ARN ou de l'ADN guide contenant une séquence pour le coder, et/ou une nucléase guidée par l'ARN, ou des acides nucléiques comprenant une séquence qui les code. Dans l'expression (I), n est un nombre entier d'une valeur de 2 à 5, R est un groupe alkyle en C1-5 linéaire, un groupe alcényle en C7-11 linéaire ou un groupe alcanediényle en C11 linéaire, les lignes ondulées représentent indépendamment des liaisons cis ou trans.
(JA) 本発明は、細胞における高い遺伝子改変効率の実現が可能な遺伝子改変ツールを送達するためのデリバリー手法を提供する。本発明に係る組成物は、1)式(I)で表される化合物又はその塩、2)構造脂質、ならびに3)ガイドRNAもしくはこれをコードする配列を含むDNA、及び/又はRNA誘導型ヌクレアーゼもしくはこれをコードする配列を含む核酸を含む、細胞中の標的遺伝子座における遺伝子改変を誘導するための組成物である。式(I)中、nは、2~5の整数を、Rは、直鎖状C1-5アルキル基、直鎖状C7-11アルケニル基又は直鎖状C11アルカジエニル基を、波線は、それぞれ独立して、シス型またはトランス型の結合を示す。
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