AU2018254410B2 - Optimization of enzyme replacement therapy for treatment of homocystinuria - Google Patents

Optimization of enzyme replacement therapy for treatment of homocystinuria Download PDF

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Publication number
AU2018254410B2
AU2018254410B2 AU2018254410A AU2018254410A AU2018254410B2 AU 2018254410 B2 AU2018254410 B2 AU 2018254410B2 AU 2018254410 A AU2018254410 A AU 2018254410A AU 2018254410 A AU2018254410 A AU 2018254410A AU 2018254410 B2 AU2018254410 B2 AU 2018254410B2
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Prior art keywords
ooe
htcbs
peg
pegylation
nhs ester
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AU2018254410A
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AU2018254410A1 (en
Inventor
Erez M BUBLIL
Frank GLAVIN
Jan P Kraus
Tomas Majtan
Marcia Sellos-Moura
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.)
Travere Therapeutics Switzerland GmbH
University of Colorado Colorado Springs
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Travere Therapeutics Switzerland GmbH
University of Colorado System
University of Colorado Colorado Springs
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Application filed by Travere Therapeutics Switzerland GmbH, University of Colorado System, University of Colorado Colorado Springs filed Critical Travere Therapeutics Switzerland GmbH
Publication of AU2018254410A1 publication Critical patent/AU2018254410A1/en
Assigned to THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE, TRAVERE THERAPEUTICS SWITZERLAND GMBH reassignment THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE Amend patent request/document other than specification (104) Assignors: ORPHAN TECHNOLOGIES, LTD., THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE
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    • 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
    • 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)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • 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)
AU2018254410A 2017-04-17 2018-04-17 Optimization of enzyme replacement therapy for treatment of homocystinuria Active AU2018254410B2 (en)

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

Publications (2)

Publication Number Publication Date
AU2018254410A1 AU2018254410A1 (en) 2019-10-10
AU2018254410B2 true AU2018254410B2 (en) 2025-02-06

Family

ID=63856394

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018254410A Active AU2018254410B2 (en) 2017-04-17 2018-04-17 Optimization of enzyme replacement therapy for treatment of homocystinuria

Country Status (9)

Country Link
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

Citations (2)

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WO2014120770A1 (en) * 2013-01-29 2014-08-07 The Regents Of University Of Colorado Cystathionine beta-synthase enzyme for treatment of homocystinuria
WO2017083327A1 (en) * 2015-11-09 2017-05-18 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for treatment of homocystinuria

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JPS57209206A (en) 1981-06-08 1982-12-22 Kanebo Ltd Skin type pack agent
US5656425A (en) 1993-08-17 1997-08-12 Regents Of The University Of Colorado Screening for mutations by expressing cDNA segments
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
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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
CA2217696A1 (en) 1995-04-25 1996-10-31 Oridigm Corporation S-adenosyl methionine regulation of metabolic pathways and its use in diagnosis and therapy
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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
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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

Patent Citations (2)

* Cited by examiner, † Cited by third party
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WO2014120770A1 (en) * 2013-01-29 2014-08-07 The Regents Of University Of Colorado Cystathionine beta-synthase enzyme for treatment of homocystinuria
WO2017083327A1 (en) * 2015-11-09 2017-05-18 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for treatment of homocystinuria

Also Published As

Publication number Publication date
CA3059592A1 (en) 2018-10-25
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
JP7146897B2 (ja) 2022-10-04
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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Owner name: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE

Free format text: FORMER NAME(S): THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE; ORPHAN TECHNOLOGIES, LTD.

Owner name: TRAVERE THERAPEUTICS SWITZERLAND GMBH

Free format text: FORMER NAME(S): THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE; ORPHAN TECHNOLOGIES, LTD.

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