ZA201601236B - Modulators of complement factor b - Google Patents

Modulators of complement factor b

Info

Publication number
ZA201601236B
ZA201601236B ZA2016/01236A ZA201601236A ZA201601236B ZA 201601236 B ZA201601236 B ZA 201601236B ZA 2016/01236 A ZA2016/01236 A ZA 2016/01236A ZA 201601236 A ZA201601236 A ZA 201601236A ZA 201601236 B ZA201601236 B ZA 201601236B
Authority
ZA
South Africa
Prior art keywords
modulators
complement factor
complement
factor
Prior art date
Application number
ZA2016/01236A
Other languages
English (en)
Inventor
Susan M Freier
Andrew T Watt
Michael L Mccaleb
Tamar R Grossman
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of ZA201601236B publication Critical patent/ZA201601236B/en

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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
    • 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
    • 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
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Urology & Nephrology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Peptides Or Proteins (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Epidemiology (AREA)
  • Optical Communication System (AREA)
  • Amplifiers (AREA)
ZA2016/01236A 2013-09-13 2016-02-22 Modulators of complement factor b ZA201601236B (en)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
ZA201601236B true ZA201601236B (en) 2017-05-31

Family

ID=52666522

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2016/01236A ZA201601236B (en) 2013-09-13 2016-02-22 Modulators of complement factor b

Country Status (32)

Country Link
US (5) US20160222389A1 (enExample)
EP (2) EP3043827B1 (enExample)
JP (3) JP6666250B2 (enExample)
KR (1) KR102381790B1 (enExample)
CN (2) CN105744959B (enExample)
AU (2) AU2014318580B2 (enExample)
BR (1) BR112016004671B1 (enExample)
CA (1) CA2921842A1 (enExample)
CL (2) CL2016000606A1 (enExample)
CR (1) CR20160170A (enExample)
DK (1) DK3043827T3 (enExample)
DO (1) DOP2016000063A (enExample)
EA (1) EA035433B1 (enExample)
ES (1) ES2745758T3 (enExample)
HR (1) HRP20191600T1 (enExample)
HU (1) HUE045109T2 (enExample)
IL (1) IL244095A0 (enExample)
LT (1) LT3043827T (enExample)
MA (1) MA38959A1 (enExample)
MX (2) MX371518B (enExample)
MY (1) MY181251A (enExample)
PE (2) PE20190354A1 (enExample)
PH (1) PH12016500443A1 (enExample)
PL (1) PL3043827T3 (enExample)
PT (1) PT3043827T (enExample)
RS (1) RS59252B1 (enExample)
RU (1) RU2662967C2 (enExample)
SG (2) SG10201806787VA (enExample)
SI (1) SI3043827T1 (enExample)
UA (1) UA119046C2 (enExample)
WO (1) WO2015038939A2 (enExample)
ZA (1) ZA201601236B (enExample)

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CR20160554A (es) * 2014-05-01 2017-03-07 Ionis Pharmaceuticals Inc Composiciones y métodos para modular la expresión del factor b del complemento
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CA3078971A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
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CA3158320A1 (en) 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
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MX2022013606A (es) * 2020-04-30 2023-01-16 Alnylam Pharmaceuticals Inc Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas.
KR20230049115A (ko) 2020-08-07 2023-04-12 상하이 메이유에 바이오테크 디벨롭먼트 컴퍼니 리미티드 보체 인자 b 억제제 및 이의 약학적 조성물, 제조 방법 및 용도
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
EP4373940A4 (en) 2021-07-17 2025-10-01 Sirnaomics Inc PRODUCTS AND COMPOSITIONS
US20250136977A1 (en) 2021-09-02 2025-05-01 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
AU2022376760A1 (en) 2021-10-27 2024-05-02 Hansoh Bio Llc Piperidinyl indole derivatives, preparation methods and medicinal uses thereof
AR127477A1 (es) * 2021-10-29 2024-01-31 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA EL FACTOR B DEL COMPLEMENTO (CFB) Y MÉTODOS DE USO DE LAS MISMAS
EP4437104A4 (en) 2021-11-24 2026-01-21 Adarx Pharmaceuticals Inc COMPLEMENT FACTOR B MODULATION COMPOSITIONS AND THEIR METHODS OF USE
WO2023129496A2 (en) * 2021-12-27 2023-07-06 Apellis Pharmaceuticals, Inc. Rnas for complement inhibition
EP4471021A4 (en) 2022-01-26 2025-04-30 Shanghai Meiyue Biotech Development Co., Ltd. Salt type and crystal form of complement factor b inhibitor, and preparation method therefor and application thereof
JP2025530222A (ja) 2022-09-10 2025-09-11 江▲蘇▼豪森▲薬▼▲業▼集▲団▼有限公司 2-置換ピペリジン誘導体、その製造方法及び医薬的用途
AU2023372971A1 (en) * 2022-11-02 2025-05-15 Ionis Pharmaceuticals, Inc. Methods for modulating complement factor b expression
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