HRP20191600T1 - Modulatori faktora b komplementa - Google Patents
Modulatori faktora b komplementa Download PDFInfo
- Publication number
- HRP20191600T1 HRP20191600T1 HRP20191600T HRP20191600T1 HR P20191600 T1 HRP20191600 T1 HR P20191600T1 HR P20191600 T HRP20191600 T HR P20191600T HR P20191600 T1 HRP20191600 T1 HR P20191600T1
- Authority
- HR
- Croatia
- Prior art keywords
- segment
- wing
- compound
- linked nucleosides
- modified oligonucleotide
- Prior art date
Links
- 102000003712 Complement factor B Human genes 0.000 title 1
- 108090000056 Complement factor B Proteins 0.000 title 1
- 239000002777 nucleoside Substances 0.000 claims 15
- 150000001875 compounds Chemical class 0.000 claims 13
- 125000003835 nucleoside group Chemical group 0.000 claims 12
- 108091034117 Oligonucleotide Proteins 0.000 claims 10
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 8
- 125000006850 spacer group Chemical group 0.000 claims 8
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 4
- 229940104302 cytosine Drugs 0.000 claims 4
- 206010064930 age-related macular degeneration Diseases 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 208000002780 macular degeneration Diseases 0.000 claims 3
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 3
- 208000035913 Atypical hemolytic uremic syndrome Diseases 0.000 claims 2
- 208000016323 C3 glomerulonephritis Diseases 0.000 claims 2
- 208000004451 Membranoproliferative Glomerulonephritis Diseases 0.000 claims 2
- 230000024203 complement activation Effects 0.000 claims 2
- 208000022401 dense deposit disease Diseases 0.000 claims 2
- 208000017169 kidney disease Diseases 0.000 claims 2
- 201000000596 systemic lupus erythematosus Diseases 0.000 claims 2
- 102100035325 Complement factor H-related protein 5 Human genes 0.000 claims 1
- 208000008069 Geographic Atrophy Diseases 0.000 claims 1
- 101000878134 Homo sapiens Complement factor H-related protein 5 Proteins 0.000 claims 1
- 208000005777 Lupus Nephritis Diseases 0.000 claims 1
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 208000011325 dry age related macular degeneration Diseases 0.000 claims 1
- 230000008482 dysregulation Effects 0.000 claims 1
- 230000000116 mitigating effect Effects 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
- C12N15/1137—Non-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 against enzymes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21047—Alternative-complement-pathway C3/C5 convertase (3.4.21.47), i.e. properdin factor B
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
Claims (12)
1. Spoj koji obuhvaća modificiran oligonukleotid koji se sastoji od 20 vezanih nukleozida koji imaju nukleobaznu sekvencu koja obuhvaća sekvencu koja je navedena u SEQ ID NO: 440, 198, 228, 237, 444, 448, 450, 453, ili 455, pri čemu modificiran oligonukleotid obuhvaća:
segment razmaka koji se sastoji od deset vezanih deoksinukleozida;
5’ segment „krila“ koji se sastoji od pet vezanih nukleozida; i
3’ segment „krila“ koji se sastoji od pet vezanih nukleozida;
pri čemu se segment razmaka nalazi između 5’ segmenta „krila“ i 3’ segmenta „krila“, pri čemu svaki nukleozid iz svakog segmenta „krila“ obuhvaća 2’-O-metoksietil šećer; pri čemu svaka internukleozidna veza je fosforotioatna veza i pri čemu svaki citozin je 5-metilcitozin.
2. Spoj prema zahtjevu 1, pri čemu se modificiran oligonukleotid sastoji od 20 vezanih nukleozida koji imaju nukleobaznu sekvencu koja se sastoji od sekvence koja je navedena u SEQ ID NO: 440, pri čemu modificiran oligonukleotid obuhvaća:
segment razmaka koji se sastoji od deset vezanih deoksinukleozida;
5’ segment „krila“ koji se sastoji od pet vezanih nukleozida; i
3’ segment „krila“ koji se sastoji od pet vezanih nukleozida;
pri čemu se segment razmaka nalazi između 5’ segmenta „krila“ i 3’ segmenta „krila“, pri čemu svaki nukleozid iz svakog segmenta „krila“ obuhvaća 2’-O-metoksietil šećer; pri čemu svaka internukleozidna veza je fosforotioatna veza i pri čemu svaki citozin je 5-metilcitozin.
3. Spoj koji ima formulu:
ili njegova farmaceutski prihvatljiva sol.
4. Spoj koji obuhvaća modificiran oligonukleotid koji se sastoji od 16 vezanih nukleozida koji imaju nukleobaznu sekvencu koja se sastoji od sekvence koja je navedena u SEQ ID NO: 598, pri čemu modificiran oligonukleotid obuhvaća:
segment razmaka koji se sastoji od deset vezanih dezoksinukleozida;
5’ segment „krila“ koji se sastoji od tri vezana nukleozida; i
3’ segment „krila“ koji se sastoji od tri vezana nukleozida;
pri čemu se segment razmaka nalazi između 5’ segmenta „krila“ i 3’ segmenta„krila“; pri čemu 5’ segment „krila“ obuhvaća 2’-O-metoksietil šećer, 2’-O-metoksietil šećer, i cEt šećer u 5’ do 3’ smjeru; pri čemu 3’ segment „krila“ obuhvaća cEt šećer, cEt šećer, i 2’-O-metoksietil šećer u 5’ do 3’ smjeru; pri čemu svaka internukleozidna veza je fosforotioatna veza; i pri čemu svaki citozin je 5-metilcitozin.
5. Spoj koji obuhvaća modificiran oligonukleotid koji se sastoji od 16 vezanih nukleozida koji imaju nukleobaznu sekvencu koja obuhvaća sekvencu koja je navedena u SEQ ID NO: 549, pri čemu modificiran oligonukleotid obuhvaća:
segment razmaka koji se sastoji od deset vezanih deoksinukleozida;
5’ segment „krila“ koji se sastoji od tri vezana nukleozida; i
3’ segment „krila“ koji se sastoji od tri vezana nukleozida;
pri čemu se segment razmaka nalazi između 5’ segmenta „krila“ i 3’ segmenta„krila“; pri čemu svaki nukleozid iz svakog segmenta „krila“ obuhvaća cEt šećer; pri čemu svaka internukleozidna veza je fosforotioatna veza; i pri čemu svaki citozin je 5-metilcitozin.
6. Spoj prema bilo kojem od zahtjeva 1 do 5, pri čemu se spoj sastoji od modificiranog oligonukleotida.
7. Spoj prema bilo kojem od zahtjeva 1 do 6, pri čemu modificiran oligonukleotid dalje obuhvaća konjugiranu grupu.
8. Sastav koji obuhvaća spoj prema bilo kojem od zahtjeva 1 do 7 i farmaceutski prihvatljiv nosač.
9. Spoj prema bilo kojem od zahtjeva 1 do 7, ili kompozicija prema zahtjevu 8, za upotrebu u liječenju, prevenciji, ili ublažavanju bolesti koja je povezana s disregulacijom alternativnog puta komplementa.
10. Spoj ili sastav za upotrebu prema zahtjevu 9, pri čemu je alternativni put komplementa aktiviran više od normalnog.
11. Spoj ili sastav za upotrebu prema zahtjevu 9, pri čemu je bolest makularna degeneracija, starosna makularna degeneracija (AMD), vlažna AMD, suha AMD, ili geografska atrofija.
12. Spoj ili sastav za upotrebu prema zahtjevu 9, pri čemu je bolest bolest bubrega, lupus nefritis, sistemski eritemski lupus (SLE), bolest gustih depozita (DDD), C3 glomerulonefritis (C3GN), CFHR5 nefropatija, ili atipični hemolitički uremijski sindrom (aHUS).
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361877624P | 2013-09-13 | 2013-09-13 | |
PCT/US2014/055458 WO2015038939A2 (en) | 2013-09-13 | 2014-09-12 | Modulators of complement factor b |
EP14844168.6A EP3043827B1 (en) | 2013-09-13 | 2014-09-12 | Modulators of complement factor b |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20191600T1 true HRP20191600T1 (hr) | 2019-11-29 |
Family
ID=52666522
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20191600 HRP20191600T1 (hr) | 2013-09-13 | 2019-09-04 | Modulatori faktora b komplementa |
Country Status (33)
Country | Link |
---|---|
US (4) | US20160222389A1 (hr) |
EP (2) | EP3043827B1 (hr) |
JP (3) | JP6666250B2 (hr) |
KR (1) | KR102381790B1 (hr) |
CN (2) | CN112359042A (hr) |
AU (2) | AU2014318580B2 (hr) |
BR (1) | BR112016004671B1 (hr) |
CA (1) | CA2921842A1 (hr) |
CL (2) | CL2016000606A1 (hr) |
CR (1) | CR20160170A (hr) |
DK (1) | DK3043827T3 (hr) |
DO (1) | DOP2016000063A (hr) |
EA (1) | EA035433B1 (hr) |
ES (1) | ES2745758T3 (hr) |
HK (1) | HK1225638A1 (hr) |
HR (1) | HRP20191600T1 (hr) |
HU (1) | HUE045109T2 (hr) |
IL (1) | IL244095A0 (hr) |
LT (1) | LT3043827T (hr) |
MA (1) | MA38959A1 (hr) |
MX (2) | MX371518B (hr) |
MY (1) | MY181251A (hr) |
PE (2) | PE20190354A1 (hr) |
PH (1) | PH12016500443A1 (hr) |
PL (1) | PL3043827T3 (hr) |
PT (1) | PT3043827T (hr) |
RS (1) | RS59252B1 (hr) |
RU (1) | RU2662967C2 (hr) |
SG (2) | SG10201806787VA (hr) |
SI (1) | SI3043827T1 (hr) |
UA (1) | UA119046C2 (hr) |
WO (1) | WO2015038939A2 (hr) |
ZA (1) | ZA201601236B (hr) |
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DK2092065T4 (da) | 2006-10-18 | 2019-10-21 | Ionis Pharmaceuticals Inc | Antisense-forbindelser |
US10017764B2 (en) | 2011-02-08 | 2018-07-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
DK2992098T3 (da) | 2013-05-01 | 2019-06-17 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression |
BR112016004671B1 (pt) | 2013-09-13 | 2020-12-29 | Ionis Pharmaceuticals, Inc. | compostos moduladores do fator b do complemento, composição compreendendo os referidos compostos e usos dos compostos ou da composição |
BR112016013148B1 (pt) | 2013-12-12 | 2024-02-27 | Alnylam Pharmaceuticals, Inc | ÁCIDO RIBONUCLEICO DE FITA DUPLA (dsRNA) PARA INIBIÇÃO DA EXPRESSÃO DO FATOR B DO COMPLEMENTO (CFB) EM UMA CÉLULA, COMPOSIÇÃO FARMACÊUTICA PARA INIBIÇÃO DA EXPRESSÃO DE UM GENE DO FATOR B DO COMPLEMENTO, SEU USO, E MÉTODO IN VITRO DE INIBIÇÃO DA EXPRESSÃO DO FATOR B DO COMPLEMENTO (CFB) EM UMA CÉLULA |
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TW201730341A (zh) * | 2016-02-05 | 2017-09-01 | Kyowa Hakko Kirin Co Ltd | 抑制補體b因子之表現之反義寡核苷酸 |
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CN110177544A (zh) | 2016-11-29 | 2019-08-27 | 普尔泰克健康有限公司 | 用于递送治疗剂的外泌体 |
WO2018117253A1 (ja) * | 2016-12-23 | 2018-06-28 | 協和発酵キリン株式会社 | 補体b因子の発現を抑制する核酸 |
WO2019027015A1 (ja) * | 2017-08-02 | 2019-02-07 | 協和発酵キリン株式会社 | 核酸複合体 |
CA3078971A1 (en) | 2017-11-01 | 2019-05-09 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof |
WO2020109343A1 (en) | 2018-11-29 | 2020-06-04 | F. Hoffmann-La Roche Ag | Combination therapy for treatment of macular degeneration |
KR20230017789A (ko) * | 2020-04-30 | 2023-02-06 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 인자 B (CFB) iRNA 조성물 및 이의 사용 방법 |
CN114057758A (zh) | 2020-08-07 | 2022-02-18 | 上海美悦生物科技发展有限公司 | 补体因子b抑制剂及其药物组合物、制备方法和用途 |
CN114057692B (zh) | 2020-08-07 | 2023-07-21 | 上海美悦生物科技发展有限公司 | 一种杂环类化合物、其制备方法及用途 |
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AU2022336157A1 (en) | 2021-09-02 | 2024-03-14 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell |
WO2023076451A1 (en) * | 2021-10-29 | 2023-05-04 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
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