CN114096250B - 红细胞生成性原卟啉症(epp)和x连锁原卟啉症(xlp)的疗法 - Google Patents

红细胞生成性原卟啉症(epp)和x连锁原卟啉症(xlp)的疗法 Download PDF

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CN114096250B
CN114096250B CN202080049199.0A CN202080049199A CN114096250B CN 114096250 B CN114096250 B CN 114096250B CN 202080049199 A CN202080049199 A CN 202080049199A CN 114096250 B CN114096250 B CN 114096250B
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CN114096250A (zh
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马小超
朱骏杰
吕杰
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University of Pittsburgh
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/12—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains three hetero rings
    • C07D471/14—Ortho-condensed systems
    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03044—Xenobiotic-transporting ATPase (3.6.3.44)

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Peptides Or Proteins (AREA)
CN202080049199.0A 2019-05-20 2020-05-20 红细胞生成性原卟啉症(epp)和x连锁原卟啉症(xlp)的疗法 Active CN114096250B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962850061P 2019-05-20 2019-05-20
US62/850,061 2019-05-20
PCT/US2020/033746 WO2020236901A1 (en) 2019-05-20 2020-05-20 A novel therapy for erythropoietic protoporphyria (epp) and x-linked protoporphyria (xlp)

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CN114096250A CN114096250A (zh) 2022-02-25
CN114096250B true CN114096250B (zh) 2025-01-14

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CN202080049199.0A Active CN114096250B (zh) 2019-05-20 2020-05-20 红细胞生成性原卟啉症(epp)和x连锁原卟啉症(xlp)的疗法

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US (1) US12528808B2 (https=)
EP (1) EP3972605A4 (https=)
JP (2) JP7820798B2 (https=)
CN (1) CN114096250B (https=)
CA (1) CA3135592A1 (https=)
WO (1) WO2020236901A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3135592A1 (en) 2019-05-20 2020-11-26 Xiaochao Ma Therapeutic agents for erythropoietic protoporphyria (epp) and x-linked protoporphyria (xlp)
US11813257B2 (en) * 2020-01-09 2023-11-14 Disc Medicine, Inc. Methods of treating erythropoietic protoporphyria, X-linked protoporphyria, or congenital erythropoietic porphyria with glycine transport inhibitors
CN120965522B (zh) * 2025-10-20 2026-02-17 兰树化妆品股份有限公司 一种Fmoc-Tea-OH及其制备方法和在多肽合成中的应用

Citations (2)

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CN106191061A (zh) * 2016-07-18 2016-12-07 暨南大学 一种特异靶向人ABCG2基因的sgRNA导向序列及其应用
WO2019060742A1 (en) * 2017-09-22 2019-03-28 Kymera Therapeutics, Inc AGENTS FOR DEGRADING PROTEINS AND USES THEREOF

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US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US4704692A (en) 1986-09-02 1987-11-03 Ladner Robert C Computer based system and method for determining and displaying possible chemical structures for converting double- or multiple-chain polypeptides to single-chain polypeptides
DE69120146T2 (de) 1990-01-12 1996-12-12 Cell Genesys Inc Erzeugung xenogener antikörper
DE69133557D1 (de) 1990-08-29 2007-03-15 Pharming Intellectual Pty Bv Homologe rekombination in säugetier-zellen
US5565332A (en) 1991-09-23 1996-10-15 Medical Research Council Production of chimeric antibodies - a combinatorial approach
US5652138A (en) 1992-09-30 1997-07-29 The Scripps Research Institute Human neutralizing monoclonal antibodies to human immunodeficiency virus
ATE183513T1 (de) 1993-06-03 1999-09-15 Therapeutic Antibodies Inc Herstellung von antikörperfragmenten
US6180377B1 (en) 1993-06-16 2001-01-30 Celltech Therapeutics Limited Humanized antibodies
US6130364A (en) 1995-03-29 2000-10-10 Abgenix, Inc. Production of antibodies using Cre-mediated site-specific recombination
US6410690B1 (en) 1995-06-07 2002-06-25 Medarex, Inc. Therapeutic compounds comprised of anti-Fc receptor antibodies
FR2765243B1 (fr) 1997-06-30 1999-07-30 Usinor Acier inoxydable austenoferritique a tres bas nickel et presentant un fort allongement en traction
US9114102B2 (en) 2007-11-07 2015-08-25 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Method of inhibiting ABCG2 and related treatments
WO2009088831A2 (en) 2008-01-03 2009-07-16 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Method of inhibiting abcg2 and other treatment methods
US9056111B1 (en) 2011-09-21 2015-06-16 Stc.Unm Selective efflux inhibitors and related pharmaceutical compositions and methods of treatment
EP3054951A4 (en) * 2013-10-07 2017-05-10 Millennium Pharmaceuticals, Inc. Inhibitor of breast cancer resistance protein (bcrp)
WO2016004043A1 (en) * 2014-06-30 2016-01-07 Blend Therapeutics, Inc. Targeted conjugates and particles and formulations thereof
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CA3135592A1 (en) 2019-05-20 2020-11-26 Xiaochao Ma Therapeutic agents for erythropoietic protoporphyria (epp) and x-linked protoporphyria (xlp)

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Publication number Priority date Publication date Assignee Title
CN106191061A (zh) * 2016-07-18 2016-12-07 暨南大学 一种特异靶向人ABCG2基因的sgRNA导向序列及其应用
WO2019060742A1 (en) * 2017-09-22 2019-03-28 Kymera Therapeutics, Inc AGENTS FOR DEGRADING PROTEINS AND USES THEREOF

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
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Publication number Publication date
EP3972605A1 (en) 2022-03-30
JP2026027223A (ja) 2026-02-18
CA3135592A1 (en) 2020-11-26
US12528808B2 (en) 2026-01-20
WO2020236901A1 (en) 2020-11-26
CN114096250A (zh) 2022-02-25
US20220249473A1 (en) 2022-08-11
JP7820798B2 (ja) 2026-02-26
EP3972605A4 (en) 2023-10-04
JP2022532923A (ja) 2022-07-20

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