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1. (WO2018003739) NUCLEIC ACID COMPOSITE INCLUDING FUNCTIONAL LIGAND
Latest bibliographic data on file with the International BureauSubmit observation

Pub. No.: WO/2018/003739 International Application No.: PCT/JP2017/023387
Publication Date: 04.01.2018 International Filing Date: 26.06.2017
IPC:
C12N 15/09 (2006.01) ,A61K 31/713 (2006.01) ,A61K 48/00 (2006.01) ,A61P 35/00 (2006.01) ,C07H 21/04 (2006.01) ,C12N 15/113 (2010.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
713
Double-stranded nucleic acids or oligonucleotides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35
Antineoplastic agents
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21
Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
04
with deoxyribosyl as saccharide radical
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Applicants:
レナセラピューティクス株式会社 RENA THERAPEUTICS INC. [JP/JP]; 東京都千代田区大手町一丁目9番2号 9-2, Otemachi 1-chome, Chiyoda-Ku, Tokyo 1000004, JP
Inventors:
矢野 純一 YANO Junichi; JP
谷川原 万顕 TANIGAWARA Kazuaki; JP
秋澤 拓也 AKISAWA Takuya; JP
Agent:
江崎 光史 ESAKI Koushi; JP
鍛冶澤 實 KAJISAWA Makoto; JP
上西 克礼 KAMINISHI Yoshinori; JP
虎山 一郎 TORAYAMA Ichiro; JP
Priority Data:
2016-13079830.06.2016JP
Title (EN) NUCLEIC ACID COMPOSITE INCLUDING FUNCTIONAL LIGAND
(FR) COMPOSITE D'ACIDE NUCLÉIQUE COMPRENANT UN LIGAND FONCTIONNEL
(JA) 機能的リガンドを含む核酸複合体
Abstract:
(EN) To provide a nucleic acid composite, preferably a double-stranded nucleic acid composite, having an excellent target gene expression-suppressing effect. The above problem is overcome by using a nucleic acid composite, preferably a double-stranded nucleic acid composite, including an active moiety which is complementary to a transcript (for example, a transcript of a target gene) and includes a DNA-based antisense nucleic acid, and a carrier moiety including a nucleic acid complementary to said nucleic acid, at least one ligand being bonded to a specific region of the nucleic acid in the carrier moiety.
(FR) L'invention vise à fournir un composite d'acide nucléique, de préférence un composite d'acide nucléique double brin, présentant un excellent effet de suppression de l'expression d'un gène cible. Le problème ci-dessus est résolu grâce à l'utilisation d'un composite d'acide nucléique, de préférence d'un composite d'acide nucléique double brin, comprenant une fraction active qui est complémentaire d'un produit de transcription (par exemple, un produit de transcription d'un gène cible) et comprend un acide nucléique antisens à base d'ADN, et une fraction support comprenant un acide nucléique complémentaire dudit acide nucléique, au moins un ligand étant lié à une région spécifique de l'acide nucléique dans la fraction support.
(JA) 優れた標的遺伝子発現抑制効果を有する核酸複合体、好ましくは二本鎖核酸複合体を提供すること。 転写産物(例えば標的遺伝子の転写産物)に相補的でありかつDNAをベースとするアンチセンス核酸を含む活性部分と、該核酸に相補的である核酸を含むキャリアー部分とを含み、さらに、少なくとも1つのリガンドが、上記キャリアー部分の核酸の特定の領域に結合されている核酸複合体、好ましくは二本鎖核酸複合体を用いることにより、上記課題は解決される。
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: Japanese (JA)
Filing Language: Japanese (JA)