CN121628883A - 用于治疗高胱氨酸尿症的胱硫醚β-合酶 - Google Patents

用于治疗高胱氨酸尿症的胱硫醚β-合酶

Info

Publication number
CN121628883A
CN121628883A CN202511736838.1A CN202511736838A CN121628883A CN 121628883 A CN121628883 A CN 121628883A CN 202511736838 A CN202511736838 A CN 202511736838A CN 121628883 A CN121628883 A CN 121628883A
Authority
CN
China
Prior art keywords
cbs
rhcbs
pegylated
enzyme
delta
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202511736838.1A
Other languages
English (en)
Chinese (zh)
Inventor
J·P·克劳斯
T·马吉坦
E·巴伯李尔
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Colorado Boulder
Original Assignee
University of Colorado Boulder
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 University of Colorado Boulder filed Critical University of Colorado Boulder
Publication of CN121628883A publication Critical patent/CN121628883A/zh
Pending legal-status Critical Current

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Classifications

    • 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
    • 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
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
    • A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
    • 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
    • A61K31/205—Amine addition salts of organic acids; Inner quaternary ammonium salts, e.g. betaine, carnitine
    • 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
    • A61K31/38—Heterocyclic compounds having sulfur as a ring hetero atom
    • A61K31/385—Heterocyclic compounds having sulfur as a ring hetero atom having two or more sulfur atoms in the same ring
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • 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
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Neurosurgery (AREA)
  • Vascular Medicine (AREA)
CN202511736838.1A 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶 Pending CN121628883A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201361758138P 2013-01-29 2013-01-29
US61/758138 2013-01-29
US13/803804 2013-03-14
US13/803,804 US9034318B2 (en) 2011-08-30 2013-03-14 Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
PCT/US2014/013602 WO2014120770A1 (en) 2013-01-29 2014-01-29 Cystathionine beta-synthase enzyme for treatment of homocystinuria
CN201480006554.0A CN105452457A (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201480006554.0A Division CN105452457A (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶

Publications (1)

Publication Number Publication Date
CN121628883A true CN121628883A (zh) 2026-03-10

Family

ID=51223166

Family Applications (3)

Application Number Title Priority Date Filing Date
CN202511736838.1A Pending CN121628883A (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶
CN201480006554.0A Pending CN105452457A (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶
CN202011103747.1A Active CN112574982B (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶

Family Applications After (2)

Application Number Title Priority Date Filing Date
CN201480006554.0A Pending CN105452457A (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶
CN202011103747.1A Active CN112574982B (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶

Country Status (12)

Country Link
US (4) US9034318B2 (https=)
EP (4) EP3712264B1 (https=)
JP (4) JP6453243B2 (https=)
CN (3) CN121628883A (https=)
AU (3) AU2014212471B2 (https=)
BR (1) BR112015017775A2 (https=)
DK (1) DK3712264T5 (https=)
FI (1) FI3712264T3 (https=)
HK (1) HK1213291A1 (https=)
IL (1) IL239783B (https=)
PT (1) PT3712264T (https=)
WO (1) WO2014120770A1 (https=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003251550A1 (en) 2002-06-17 2003-12-31 The Regents Of The University Of Colorado HUMAN CYSTATHIONINE Beta-SYNTHASE VARIANTS AND METHODS OF PRODU CTION THEREOF
US9034318B2 (en) * 2011-08-30 2015-05-19 The Regents Of The University Of Colorado, A Body Corporate Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
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
EP3456700A1 (en) 2013-10-18 2019-03-20 Deutsches Krebsforschungszentrum Labeled inhibitors of prostate specific membrane antigen (psma), their use as imaging agents and pharmaceutical agents for the treatment of prostate cancer
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
KR102680776B1 (ko) 2017-05-12 2024-07-04 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 고호모시스테인혈증 및 호모시스틴뇨증 환자를 치료하기 위한 인간-효소 매개된 호모시스테인의 고갈
WO2019183294A1 (en) 2018-03-20 2019-09-26 Rubius Therapeutics, Inc. Therapeutic cell systems and methods for treating homocystinuria
CN108795913B (zh) * 2018-05-04 2021-07-02 山西大学 一种植物中能催化h2s产生的酶及其应用
CN113260324A (zh) 2018-10-26 2021-08-13 得克萨斯大学体系董事会 用于治疗用途的工程化灵长目动物胱氨酸/半胱氨酸降酶
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
USD1014518S1 (en) * 2021-07-09 2024-02-13 The Regents Of The University Of Colorado, A Body Corporate Display screen or portion thereof with graphical user interface
USD1013704S1 (en) * 2021-07-09 2024-02-06 The Regents Of The University Of Colorado, A Body Corporate Display screen or portion thereof with graphical user interface
CN115721735A (zh) * 2022-10-13 2023-03-03 呈诺再生医学科技(北京)有限公司 Cbs基因在制备糖尿病视网膜病变治疗药物中的应用

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JPS57209206A (en) 1981-06-08 1982-12-22 Kanebo Ltd Skin type pack agent
US5523225A (en) 1993-09-13 1996-06-04 Regents Of The University Of Colorado DNA sequence encoding human cystathionine β-synthase
US5919455A (en) 1993-10-27 1999-07-06 Enzon, Inc. Non-antigenic branched polymer conjugates
US5730990A (en) 1994-06-24 1998-03-24 Enzon, Inc. Non-antigenic amine derived polymers and polymer conjugates
US5635375A (en) 1995-01-09 1997-06-03 Regents Of The University Of Colorado Method of increasing the yield and heme saturation of cystathione β-synthase
US6153655A (en) 1998-04-17 2000-11-28 Enzon, Inc. Terminally-branched polymeric linkers and polymeric conjugates containing the same
US6635438B2 (en) * 1999-11-02 2003-10-21 Catch, Inc. Enzymatic cycling assays for homocysteine and cystathionine
AU2003251550A1 (en) 2002-06-17 2003-12-31 The Regents Of The University Of Colorado HUMAN CYSTATHIONINE Beta-SYNTHASE VARIANTS AND METHODS OF PRODU CTION THEREOF
EP1396537A1 (en) 2002-09-04 2004-03-10 Avidis SA Treatment of elevated plasma homocysteine
CN101322840A (zh) * 2008-05-28 2008-12-17 中国人民解放军军事医学科学院卫生学环境医学研究所 一种人胱硫醚β-合成酶重组蛋白及应用
EA201370008A1 (ru) 2010-07-01 2013-06-28 Айсис Инновэйшн Лимитид Лечение когнитивных нарушений
US9034318B2 (en) * 2011-08-30 2015-05-19 The Regents Of The University Of Colorado, A Body Corporate Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
US9243239B2 (en) 2012-03-26 2016-01-26 The Regents Of The University Of Colorado, A Body Corporate Purification of cystathionine beta-synthase
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

Also Published As

Publication number Publication date
EP3712264A1 (en) 2020-09-23
JP6453243B2 (ja) 2019-01-16
DK3712264T5 (da) 2024-08-26
DK3712264T3 (da) 2024-05-06
HK1249137A1 (en) 2018-10-26
EP3712264B1 (en) 2024-04-10
BR112015017775A2 (pt) 2017-11-21
IL239783A0 (en) 2015-08-31
EP2951299A1 (en) 2015-12-09
AU2022263598B2 (en) 2025-06-05
FI3712264T3 (fi) 2024-05-07
EP4406536A3 (en) 2025-01-01
JP7240743B2 (ja) 2023-03-16
JP2016506727A (ja) 2016-03-07
US20170065687A1 (en) 2017-03-09
AU2020200719B2 (en) 2022-08-18
CN112574982B (zh) 2025-05-13
AU2014212471A8 (en) 2015-08-13
AU2014212471A1 (en) 2015-07-23
AU2020200719A1 (en) 2020-02-20
EP3282013A1 (en) 2018-02-14
EP4406536A2 (en) 2024-07-31
IL239783B (en) 2019-10-31
US20150344864A1 (en) 2015-12-03
HK1213291A1 (zh) 2016-06-30
JP2021000142A (ja) 2021-01-07
EP3282013B1 (en) 2020-05-20
JP2023062201A (ja) 2023-05-02
CN112574982A (zh) 2021-03-30
PT3712264T (pt) 2024-05-09
US9447406B2 (en) 2016-09-20
EP3712264A8 (en) 2020-11-04
US10046036B2 (en) 2018-08-14
US20180311325A1 (en) 2018-11-01
US9034318B2 (en) 2015-05-19
CA2898772A1 (en) 2014-08-07
AU2022263598A1 (en) 2022-12-15
JP7580822B2 (ja) 2024-11-12
WO2014120770A1 (en) 2014-08-07
EP2951299B1 (en) 2017-07-12
AU2014212471B2 (en) 2019-11-28
CN105452457A (zh) 2016-03-30
US10624959B2 (en) 2020-04-21
JP2019059768A (ja) 2019-04-18
US20140212403A1 (en) 2014-07-31

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