CN105764498A - 纯合家族型高胆固醇血症的治疗 - Google Patents

纯合家族型高胆固醇血症的治疗 Download PDF

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Publication number
CN105764498A
CN105764498A CN201480063803.XA CN201480063803A CN105764498A CN 105764498 A CN105764498 A CN 105764498A CN 201480063803 A CN201480063803 A CN 201480063803A CN 105764498 A CN105764498 A CN 105764498A
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salt
phenoxy
trifluoromethyl
acetic acid
ethoxy
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Pending
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CN201480063803.XA
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English (en)
Chinese (zh)
Inventor
罗伯特·L·马丁
查尔斯·A·麦克沃特
帕特里克·J·奥马拉
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Shellfish Pharmaceuticals Of Sigma
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Shellfish Pharmaceuticals Of Sigma
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Publication of CN105764498A publication Critical patent/CN105764498A/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/192—Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid 
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/4468—Non condensed piperidines, e.g. piperocaine having a nitrogen directly attached in position 4, e.g. clebopride, fentanyl
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against enzymes
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Emergency Medicine (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
CN201480063803.XA 2013-11-20 2014-11-14 纯合家族型高胆固醇血症的治疗 Pending CN105764498A (zh)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201361906837P 2013-11-20 2013-11-20
US61/906,837 2013-11-20
US201461942438P 2014-02-20 2014-02-20
US61/942,438 2014-02-20
US201461942941P 2014-02-21 2014-02-21
US61/942,941 2014-02-21
US201461974816P 2014-04-03 2014-04-03
US201461974785P 2014-04-03 2014-04-03
US201461974725P 2014-04-03 2014-04-03
US61/974,816 2014-04-03
US61/974,725 2014-04-03
US61/974,785 2014-04-03
PCT/US2014/065742 WO2015077154A1 (en) 2013-11-20 2014-11-14 Treatment of homozygous familial hypercholesterolemia

Publications (1)

Publication Number Publication Date
CN105764498A true CN105764498A (zh) 2016-07-13

Family

ID=52014370

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201480063803.XA Pending CN105764498A (zh) 2013-11-20 2014-11-14 纯合家族型高胆固醇血症的治疗

Country Status (14)

Country Link
US (1) US20150139987A1 (enExample)
EP (1) EP3071198A1 (enExample)
JP (1) JP2017505285A (enExample)
KR (1) KR20160079124A (enExample)
CN (1) CN105764498A (enExample)
AU (1) AU2014353246A1 (enExample)
CA (1) CA2930069A1 (enExample)
CL (1) CL2016001215A1 (enExample)
EA (1) EA201691039A1 (enExample)
HK (1) HK1224186A1 (enExample)
IL (1) IL245748A0 (enExample)
MX (1) MX2016006583A (enExample)
PH (1) PH12016500886A1 (enExample)
WO (1) WO2015077154A1 (enExample)

Cited By (1)

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CN113710808A (zh) * 2018-12-20 2021-11-26 宾夕法尼亚州大学信托人 用于治疗家族性高胆固醇血症的基因疗法

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US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
PT2929031T (pt) 2012-12-05 2018-01-19 Alnylam Pharmaceuticals Inc Composições de irna de pcsk9 e métodos de seu uso
US9624172B2 (en) 2014-02-17 2017-04-18 Hetero Research Foundation Polymorphs of lomitapide and its salts
DK3119384T3 (en) 2014-03-20 2018-12-03 Cymabay Therapeutics Inc TREATMENT OF INTRAHEPATHIC COLESTASTIC DISEASES
US10272058B2 (en) 2014-03-20 2019-04-30 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
UA121208C2 (uk) 2014-04-11 2020-04-27 Сімабей Терапьютікс, Інк. Лікування нажхп та насг
BR102015025502B1 (pt) * 2015-04-30 2022-06-21 Aegerion Pharmaceuticals, Inc Composição de lomitapida, tablete, produto de lomitapida, métodos para analisar uma composição de amostra de lomitapida e para determinar uma quantidade de uma impureza em uma amostra da composição
IL293355B2 (en) * 2015-08-25 2024-07-01 Alnylam Pharmaceuticals Inc Methods and compositions for treating a proprotein convertase subtilisin kexin (pcsk9) gene-associated disorder
CN105481758A (zh) * 2016-01-13 2016-04-13 天津药物研究院有限公司 一种洛美他派晶型ⅰ及其制备方法和用途
KR102456731B1 (ko) 2016-03-03 2022-10-24 리제너론 파아마슈티컬스, 인크. Pcsk9 억제제를 angptl3 억제제와 병용하여 투여함으로써 고지혈증을 갖는 환자를 치료하기 위한 방법
CA3021884A1 (en) * 2016-04-28 2017-11-02 Regeneron Pharmaceuticals, Inc. Methods for treating patients with familial hypercholesterolemia
WO2017200715A1 (en) * 2016-05-19 2017-11-23 Cymabay Therapeutics, Inc. Treatment of severe hyperlipidemia
US10512622B2 (en) 2017-07-14 2019-12-24 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
CN117085011A (zh) * 2017-11-23 2023-11-21 浙江海正药业股份有限公司 一种海泽麦布和HMG-CoA还原酶抑制剂的药物组合物
RS66416B1 (sr) 2019-01-18 2025-02-28 Astrazeneca Ab 6'-[[(1s,3s)-3-[[5-(difluorometoksi)-2-pirimidinil]amino]ciklopentil]amino][1(2h),3'-bipiridin]-2-on kao inhibitor pcsk9 i metode njegove primene
US12559551B2 (en) 2019-05-24 2026-02-24 Regeneron Pharmaceuticals, Inc. Stabilized formulations containing anti-ANGPTL3 antibodies
WO2021097034A1 (en) 2019-11-14 2021-05-20 Cymabay Therapeutics, Inc. Seladelpar for use in the treatment of intestinal barrier dysfunction and associated diseases
WO2021097027A1 (en) 2019-11-14 2021-05-20 Cymabay Therapeutics, Inc. Seladelpar for use in the treatment of alcoholic liver disease
CA3186856A1 (en) 2020-07-29 2022-02-03 Ruth NALLEN Lomitapide for use in methods of treating hyperlipidemia and hypercholesterolemia in pediatric patients

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CN113710808A (zh) * 2018-12-20 2021-11-26 宾夕法尼亚州大学信托人 用于治疗家族性高胆固醇血症的基因疗法

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Publication number Publication date
JP2017505285A (ja) 2017-02-16
KR20160079124A (ko) 2016-07-05
IL245748A0 (en) 2016-07-31
EP3071198A1 (en) 2016-09-28
AU2014353246A1 (en) 2016-06-16
CA2930069A1 (en) 2015-05-28
WO2015077154A1 (en) 2015-05-28
HK1224186A1 (zh) 2017-08-18
EA201691039A1 (ru) 2016-11-30
CL2016001215A1 (es) 2016-12-09
PH12016500886A1 (en) 2016-06-20
US20150139987A1 (en) 2015-05-21
MX2016006583A (es) 2016-09-06

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