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1. (WO2017135397) ANTISENSE OLIGONUCLEOTIDE FOR SUPPRESSING EXPRESSION OF COMPLEMENT B FACTOR
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2017/135397 International Application No.: PCT/JP2017/003904
Publication Date: 10.08.2017 International Filing Date: 03.02.2017
IPC:
C12N 15/113 (2010.01) ,A61K 31/7088 (2006.01) ,A61K 48/00 (2006.01) ,A61P 11/06 (2006.01) ,A61P 13/00 (2006.01) ,A61P 13/12 (2006.01) ,A61P 27/02 (2006.01) ,A61P 37/02 (2006.01) ,C12N 5/10 (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
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
11
Drugs for disorders of the respiratory system
06
Antiasthmatics
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13
Drugs for disorders of the urinary system
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13
Drugs for disorders of the urinary system
12
of the kidneys
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27
Drugs for disorders of the senses
02
Ophthalmic agents
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
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
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10
Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
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, Ohtemachi, Chiyoda-ku, Tokyo 1008185, JP
Inventors:
山田 陽史 YAMADA, Yoji; JP
清水 清 SHIMIZU, Kiyoshi; JP
岩井 宏徒 IWAI, Hiroto; JP
牧野 麻奈 MAKINO, Asana; JP
Agent:
高島 一 TAKASHIMA, Hajime; JP
Priority Data:
2016-02112805.02.2016JP
Title (EN) ANTISENSE OLIGONUCLEOTIDE FOR SUPPRESSING EXPRESSION OF COMPLEMENT B FACTOR
(FR) OLIGONUCLÉOTIDE ANTISENS DESTINÉ À SUPPRIMER L’EXPRESSION DU FACTEUR DU COMPLÉMENT B
(JA) 補体B因子の発現を抑制するアンチセンスオリゴヌクレオチド
Abstract:
(EN) The present invention provides: an antisense oligonucleotide having CFB expression-suppressing activity; a pharmaceutical composition including the antisense oligonucleotide; and a drug that suppresses the expression of CFB including the antisense oligonucleotide, the drug preventing or treating disorders mediated by abnormalities of the complement alternative pathway such as atypical hemolytic uremic syndrome, paroxysmal nocturnal hemoglobinuria, age-related macular degeneration, membranous-proliferative glomerulonephritis, C3 nephritis, membranous nephropathy, rapid progressive glomerulonephritis (RPGN), acute kidney injury (AKI), asthma, or autoimmune diseases.
(FR) La présente invention décrit : un oligonucléotide antisens présentant une activité d’expression-suppression de CFB ; une composition pharmaceutique comprenant l’oligonucléotide antisens ; et un médicament qui supprime l’expression de CFB incluant l’oligonucléotide antisens, le médicament prévenant ou traitant les troubles médiés par les anomalies de la voie alternative du complément telles que le syndrome urémique hémolytique atypique, l’hémoglobinurie nocturne paroxysmique, la dégénérescence maculaire liée à l’âge, la glomérulonéphrite membrano-proliférative, la néphrite C3, la néphropathie membraneuse, la glomérulonéphrite à progression rapide (RPGN), la lésion rénale aiguë (AKI), l’asthme, ou les maladies auto-immunes.
(JA) 本発明は、CFBの発現抑制活性を有するアンチセンスオリゴヌクレオチド、該アンチセンスオリゴヌクレオチドを含む医薬組成物ならびに該アンチセンスオリゴヌクレオチドを含むCFBの発現を抑制し、非典型溶血性尿毒症症候群、発作性夜間ヘモグロビン尿症、加齢黄斑変性症、膜性増殖性糸球体腎炎、C3腎炎、膜性腎症、急速進行性糸球体腎炎(RPGN)、急性腎障害(AKI)、喘息又は自己免疫疾患などの補体第二経路の異常により媒介される障害の予防または治療薬を提供する。
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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, 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)