Some content of this application is unavailable at the moment.
If this situation persist, please contact us atFeedback&Contact
1. (WO2019027015) NUCLEIC ACID COMPLEX
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2019/027015 International Application No.: PCT/JP2018/029113
Publication Date: 07.02.2019 International Filing Date: 02.08.2018
IPC:
C12N 15/113 (2010.01) ,A61K 31/713 (2006.01) ,A61K 47/54 (2017.01) ,A61K 48/00 (2006.01) ,A61P 37/02 (2006.01) ,A61P 43/00 (2006.01)
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
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
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
[IPC code unknown for A61K 47/54]
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
37
Drugs for immunological or allergic disorders
02
Immunomodulators
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43
Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
Applicants:
協和発酵キリン株式会社 KYOWA HAKKO KIRIN CO., LTD. [JP/JP]; 東京都千代田区大手町一丁目6番1号 1-6-1, Otemachi, Chiyoda-ku, Tokyo 1008185, JP
Inventors:
山田 陽史 YAMADA, Yoji; JP
牧野 麻奈 MAKINO, Asana; JP
長谷川 尚 HASEGAWA, Hisashi; JP
岩井 宏徒 IWAI, Hiroto; JP
上原 啓嗣 UEHARA, Keiji; JP
鈴木 康裕 SUZUKI, Yasuhiro; JP
Agent:
稲葉 良幸 INABA, Yoshiyuki; JP
大貫 敏史 ONUKI, Toshifumi; JP
Priority Data:
2017-14976102.08.2017JP
Title (EN) NUCLEIC ACID COMPLEX
(FR) COMPLEXE D'ACIDE NUCLÉIQUE
(JA) 核酸複合体
Abstract:
(EN) The present invention provides a nucleic acid complex represented by formula 1, the nucleic acid complex being capable of suppressing the expression of complement factor B (CFB). Formula 1: (in formula 1, X is a double-stranded nucleic acid that comprises a sense strand and an antisense strand and includes a duplex region of at least 11 base pairs; the double-stranded nucleic acid is complementary with any of the CFB mRNA sequences of interest described in Tables 1-1 to 1-3 in an oligonucleotide strand having a strand length of 17-30 nucleotides in the antisense strand, and the 3' end or 5' end of the sense strand bonds to S3; L1 and L2 each independently represent a sugar ligand; S1, S2, and S3 each independently represent a linker.)
(FR) L'invention fournit un complexe d'acide nucléique qui est représenté par la formule (1), et qui permet d'inhiber l'expression d'un facteur B de complément (CFB). Formule (1) : (Dans la formule (1), X consiste en un acide nucléique à double-brin constitué d'un brin sens et d'un brin antisens, et contenant les régions double-brin d'au moins 11 paires de base, cet acide nucléique à double-brin est complémentaire vis-à-vis de séquences ARNmCFB cibles énumérées dans les tableaux 1-1 à 1-3, dans un brin d'oligonucléotide long de 17 à 30 nucléotides dans le brin antisens, l'extrémité 3' ou l'extrémité 5' du brin sens est liée en S3, L1 et L2 représentent chacun indépendamment un ligand de sucre, et S1, S2 et S3 représentent chacun indépendamment un lieur.)
(JA) 本発明は下記式1で表される、補体B因子(CFB)の発現を抑制することが可能な核酸複合体を提供する。 式1:(式1中、 Xは、センス鎖およびアンチセンス鎖からなり、少なくとも11個の塩基対の二重鎖領域を含む二本鎖核酸であり、 該二本鎖核酸は、該アンチセンス鎖中の、17個~30個のヌクレオチドの鎖長のオリゴヌクレオチド鎖において、表1-1~表1-3に記載された標的CFB mRNA配列のいずれかと相補的であり、 該センス鎖の3'末端または5'末端はS3に結合し、 L1およびL2は、それぞれ独立して、糖リガンドであり、 S1、S2およびS3は、それぞれ独立して、リンカーである。)
front page image
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 Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (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)