ES2909914T3 - Composiciones y métodos para su uso en el tratamiento de homocistinuria - Google Patents

Composiciones y métodos para su uso en el tratamiento de homocistinuria Download PDF

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Publication number
ES2909914T3
ES2909914T3 ES16864878T ES16864878T ES2909914T3 ES 2909914 T3 ES2909914 T3 ES 2909914T3 ES 16864878 T ES16864878 T ES 16864878T ES 16864878 T ES16864878 T ES 16864878T ES 2909914 T3 ES2909914 T3 ES 2909914T3
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Prior art keywords
htcbs
peg
pegylated
mice
dose
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ES16864878T
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English (en)
Spanish (es)
Inventor
Jan Kraus
Tomas Majtan
Erez Bublil
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University of Colorado System
University of Colorado Colorado Springs
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University of Colorado System
University of Colorado Colorado Springs
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Priority claimed from US14/935,690 external-priority patent/US9675678B2/en
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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
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • 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
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/14—Drugs for dermatological disorders for baldness or alopecia
    • 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
    • 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
    • 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)
    • 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Dermatology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Urology & Nephrology (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
ES16864878T 2015-11-09 2016-11-09 Composiciones y métodos para su uso en el tratamiento de homocistinuria Active ES2909914T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US14/935,690 US9675678B2 (en) 2013-01-29 2015-11-09 Compositions and methods for treatment of homocystinuria
PCT/US2016/061050 WO2017083327A1 (en) 2015-11-09 2016-11-09 Compositions and methods for treatment of homocystinuria

Publications (1)

Publication Number Publication Date
ES2909914T3 true ES2909914T3 (es) 2022-05-10

Family

ID=58696136

Family Applications (2)

Application Number Title Priority Date Filing Date
ES16864878T Active ES2909914T3 (es) 2015-11-09 2016-11-09 Composiciones y métodos para su uso en el tratamiento de homocistinuria
ES21204193T Active ES2994256T3 (en) 2015-11-09 2016-11-09 Compositions and methods for treatment of homocystinuria

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES21204193T Active ES2994256T3 (en) 2015-11-09 2016-11-09 Compositions and methods for treatment of homocystinuria

Country Status (26)

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US (3) US11077175B2 (enExample)
EP (3) EP4487914A3 (enExample)
JP (2) JP7009364B2 (enExample)
KR (1) KR102819396B1 (enExample)
CN (2) CN115920016A (enExample)
AU (2) AU2016354030B2 (enExample)
BR (1) BR112018007768A2 (enExample)
CA (1) CA3001625A1 (enExample)
CY (1) CY1125113T1 (enExample)
DK (2) DK3998067T3 (enExample)
ES (2) ES2909914T3 (enExample)
FI (1) FI3998067T3 (enExample)
HR (2) HRP20220407T1 (enExample)
HU (2) HUE058749T2 (enExample)
IL (2) IL258487B2 (enExample)
LT (2) LT3373922T (enExample)
MX (1) MX2018005312A (enExample)
NZ (1) NZ741552A (enExample)
PL (2) PL3998067T3 (enExample)
PT (2) PT3998067T (enExample)
RS (2) RS66106B1 (enExample)
SG (1) SG10201912972SA (enExample)
SI (2) SI3998067T1 (enExample)
SM (2) SMT202400463T1 (enExample)
WO (1) WO2017083327A1 (enExample)
ZA (1) ZA201802376B (enExample)

Families Citing this family (5)

* 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
BR112018007768A2 (pt) 2015-11-09 2018-10-23 Univ Colorado Regents composições e métodos para tratamento de homocistinúria
JP7146897B2 (ja) * 2017-04-17 2022-10-04 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 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
DK3990005T3 (da) * 2019-06-26 2025-12-08 Travere Therapeutics Switzerland Gmbh Pegyleret cystathioninbetasynthase til enzymterapi til behandling af homocystinuri
EP4025243A4 (en) * 2019-09-03 2023-09-27 Travere Therapeutics Switzerland GmbH CYSTATHIONINE BETA-SYNTHASE ENZYMA THERAPY FOR THE TREATMENT OF HIGH HOMOCYSTEINE LEVELS

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BR112018007768A2 (pt) 2015-11-09 2018-10-23 Univ Colorado Regents composições e métodos para tratamento de homocistinúria
JP7146897B2 (ja) * 2017-04-17 2022-10-04 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 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
JP7002314B2 (ja) * 2017-12-13 2022-02-10 株式会社ブリヂストン 空気入りタイヤ
DK3990005T3 (da) 2019-06-26 2025-12-08 Travere Therapeutics Switzerland Gmbh Pegyleret cystathioninbetasynthase til enzymterapi til behandling af homocystinuri
EP4025243A4 (en) 2019-09-03 2023-09-27 Travere Therapeutics Switzerland GmbH CYSTATHIONINE BETA-SYNTHASE ENZYMA THERAPY FOR THE TREATMENT OF HIGH HOMOCYSTEINE LEVELS

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RS63070B1 (sr) 2022-04-29
PL3998067T3 (pl) 2025-01-07
HUE068830T2 (hu) 2025-01-28
US11077175B2 (en) 2021-08-03
JP7482914B2 (ja) 2024-05-14
AU2022287540A1 (en) 2023-02-02
CN115920016A (zh) 2023-04-07
EP4487914A3 (en) 2025-03-26
JP2018532763A (ja) 2018-11-08
EP3998067A1 (en) 2022-05-18
SMT202200151T1 (it) 2022-05-12
AU2022287540B2 (en) 2025-02-27
CA3001625A1 (en) 2017-05-18
KR20180074703A (ko) 2018-07-03
US20220125896A1 (en) 2022-04-28
NZ741552A (en) 2022-04-29
AU2016354030B2 (en) 2022-09-15
RS66106B1 (sr) 2024-11-29
PT3998067T (pt) 2024-11-04
EP4487914A2 (en) 2025-01-08
EP3373922A1 (en) 2018-09-19
HK1258502A1 (en) 2019-11-15
HRP20220407T1 (hr) 2022-05-27
DK3998067T3 (da) 2024-10-28
IL302198A (en) 2023-06-01
MX2018005312A (es) 2018-08-15
HUE058749T2 (hu) 2022-09-28
LT3998067T (lt) 2024-11-25
JP2022050589A (ja) 2022-03-30
KR102819396B1 (ko) 2025-06-12
IL258487B1 (en) 2023-05-01
HRP20241491T1 (hr) 2025-01-03
US11771745B2 (en) 2023-10-03
IL258487A (en) 2018-05-31
EP3373922B1 (en) 2022-01-05
PT3373922T (pt) 2022-03-28
ES2994256T3 (en) 2025-01-21
PL3373922T3 (pl) 2022-05-02
SI3373922T1 (sl) 2022-05-31
SMT202400463T1 (it) 2025-01-14
LT3373922T (lt) 2022-04-11
EP3373922A4 (en) 2019-05-15
US20240091325A1 (en) 2024-03-21
US20200276282A1 (en) 2020-09-03
DK3373922T3 (da) 2022-03-28
FI3998067T3 (fi) 2024-10-31
WO2017083327A1 (en) 2017-05-18
SI3998067T1 (sl) 2025-03-31
ZA201802376B (en) 2020-11-25
AU2016354030A1 (en) 2018-05-10
CN108472277A (zh) 2018-08-31
JP7009364B2 (ja) 2022-02-10
SG10201912972SA (en) 2020-03-30
CY1125113T1 (el) 2024-02-16
EP3998067B1 (en) 2024-09-11
BR112018007768A2 (pt) 2018-10-23
IL258487B2 (en) 2023-09-01
US12194082B2 (en) 2025-01-14

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