JP7146897B2 - ホモシスチン尿症の治療のための酵素補充療法の最適化 - Google Patents

ホモシスチン尿症の治療のための酵素補充療法の最適化 Download PDF

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JP7146897B2
JP7146897B2 JP2020506311A JP2020506311A JP7146897B2 JP 7146897 B2 JP7146897 B2 JP 7146897B2 JP 2020506311 A JP2020506311 A JP 2020506311A JP 2020506311 A JP2020506311 A JP 2020506311A JP 7146897 B2 JP7146897 B2 JP 7146897B2
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pegylation
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JP2020516322A (ja
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マイタン、トーマス
ピー． クラウス、ジャン
エム． バブリル、エレツ
グラビン、フランク
セロス－モウラ、マルシア
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Travere Therapeutics Switzerland GmbH
University of Colorado Colorado Springs
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University of Colorado Colorado Springs
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y402/00—Carbon-oxygen lyases (4.2)
    • C12Y402/01—Hydro-lyases (4.2.1)
    • C12Y402/01022—Cystathionine beta-synthase (4.2.1.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51—Lyases (4)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • 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
    • C12N9/88—Lyases (4.)
    • 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
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates

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  • Chemical & Material Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2020506311A 2017-04-17 2018-04-17 ホモシスチン尿症の治療のための酵素補充療法の最適化 Active JP7146897B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762486246P 2017-04-17 2017-04-17
US62/486,246 2017-04-17
PCT/US2018/027854 WO2018195006A1 (en) 2017-04-17 2018-04-17 Optimization of enzyme replacement therapy for treatment of homocystinuria

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JP2020516322A JP2020516322A (ja) 2020-06-11
JP2020516322A5 JP2020516322A5 (https=) 2021-05-27
JP7146897B2 true JP7146897B2 (ja) 2022-10-04

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US (2) US11324811B2 (https=)
EP (1) EP3612214A4 (https=)
JP (1) JP7146897B2 (https=)
CN (2) CN118064414A (https=)
AU (1) AU2018254410B2 (https=)
CA (1) CA3059592A1 (https=)
IL (1) IL269544B2 (https=)
MX (1) MX2019012347A (https=)
WO (1) WO2018195006A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9675678B2 (en) 2013-01-29 2017-06-13 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for treatment of homocystinuria
FI3998067T3 (fi) * 2015-11-09 2024-10-31 Univ Colorado Regents Koostumukset ja menetelmät homokystinurian hoitoon
IL269544B2 (en) 2017-04-17 2025-01-01 Univ Colorado Regents Streamlining enzyme replacement therapy for the treatment of high homocysteine
ES3058596T3 (en) 2019-06-26 2026-03-11 Travere Therapeutics Switzerland Gmbh Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria
CA3152379A1 (en) * 2019-09-03 2021-03-11 Travere Therapeutics Switzerland Gmbh Cystathionine beta-synthase enzyme therapy for treatment of elevated homocysteine levels
CN114965761B (zh) * 2022-05-17 2024-07-02 深圳赛保尔生物药业有限公司 聚乙二醇化蛋白药物中n-羟基琥珀酰亚胺的检测方法
WO2025193499A1 (en) * 2024-03-12 2025-09-18 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods to potentiate enzyme therapies for treating homocystinuria and homocysteine remethylation disorders

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JP2016506727A (ja) 2013-01-29 2016-03-07 ザ リージェンツ オブ ザ ユニバーシティ オブ コロラド，ア ボディー コーポレイトＴＨＥ ＲＥＧＥＮＴＳ ＯＦ ＴＨＥ ＵＮＩＶＥＲＳＩＴＹ ＯＦ ＣＯＬＯＲＡＤＯ，ａ ｂｏｄｙ ｃｏｒｐｏｒａｔｅ ホモシスチン尿症を処置するためのシスタチオニンβ−シンターゼ酵素
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CA3059592A1 (en) 2018-10-25
AU2018254410B2 (en) 2025-02-06
US12042529B2 (en) 2024-07-23
US20200261555A1 (en) 2020-08-20
US11324811B2 (en) 2022-05-10
AU2018254410A1 (en) 2019-10-10
EP3612214A4 (en) 2021-01-20
CN118064414A (zh) 2024-05-24
CN110753556A (zh) 2020-02-04
EP3612214A1 (en) 2020-02-26
IL269544A (en) 2019-11-28
JP2020516322A (ja) 2020-06-11
IL269544B2 (en) 2025-01-01
WO2018195006A1 (en) 2018-10-25
IL269544B1 (en) 2024-09-01
MX2019012347A (es) 2020-01-15
US20230039591A1 (en) 2023-02-09

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