CL2019003248A1 - Disminución de homocisteína mediada por enzimas humanas para el tratamiento de pacientes con hiperhomocisteinemia y homocistinuria. - Google Patents

Disminución de homocisteína mediada por enzimas humanas para el tratamiento de pacientes con hiperhomocisteinemia y homocistinuria.

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Publication number
CL2019003248A1
CL2019003248A1 CL2019003248A CL2019003248A CL2019003248A1 CL 2019003248 A1 CL2019003248 A1 CL 2019003248A1 CL 2019003248 A CL2019003248 A CL 2019003248A CL 2019003248 A CL2019003248 A CL 2019003248A CL 2019003248 A1 CL2019003248 A1 CL 2019003248A1
Authority
CL
Chile
Prior art keywords
homocystinuria
treatment
hyperhomocysteinemia
homocysteine
mediated
Prior art date
Application number
CL2019003248A
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English (en)
Inventor
George Georgiou
Everett Stone
Wei-Cheng Lu
Original Assignee
Univ Texas
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Publication date
Application filed by Univ Texas filed Critical Univ Texas
Publication of CL2019003248A1 publication Critical patent/CL2019003248A1/es

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    • 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
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88—Lyases (4.)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12Y404/00—Carbon-sulfur lyases (4.4)
    • C12Y404/01—Carbon-sulfur lyases (4.4.1)
    • C12Y404/01001—Cystathionine gamma-lyase (4.4.1.1)
    • 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
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    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4415—Pyridoxine, i.e. Vitamin B6
    • 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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  • Bioinformatics & Cheminformatics (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Biomedical Technology (AREA)
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  • Biochemistry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Mycology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

SE DESCRIBEN MÉTODOS Y COMPOSICIONES QUE SE REFIEREN A LA MODIFICACIÓN POR INGENIERÍA GENÉTICA DE UNA PROTEÍNA MEJORADA CON ACTIVIDAD DE ENZIMA HOMOCIST(E)INASA. POR EJEMPLO, SE DIVULGAN ENZIMAS CISTATIONINA-Γ-LIASA (CGL) MODIFICADAS QUE COMPRENDEN UNA O MÁS SUSTITUCIONES DE AMINOÁCIDOS Y CAPACES DE DEGRADAR HOMOCIST(E)ÍNA. ASIMISMO, SE PROPORCIONAN COMPOSICIONES Y MÉTODOS PARA EL TRATAMIENTO DE HOMOCISTINURIA O HIPERHOMOCISTEÍNAMIA CON DISMINUCIÓN DE HOMOCIST(E)ÍNA USANDO LAS ENZIMAS O ÁCIDOS NUCLEICOS DIVULGADOS.
CL2019003248A 2017-05-12 2019-11-11 Disminución de homocisteína mediada por enzimas humanas para el tratamiento de pacientes con hiperhomocisteinemia y homocistinuria. CL2019003248A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762505493P 2017-05-12 2017-05-12

Publications (1)

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CL2019003248A1 true CL2019003248A1 (es) 2020-03-20

Family

ID=62685099

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CL2019003248A CL2019003248A1 (es) 2017-05-12 2019-11-11 Disminución de homocisteína mediada por enzimas humanas para el tratamiento de pacientes con hiperhomocisteinemia y homocistinuria.

Country Status (13)

Country Link
US (2) US11033612B2 (es)
EP (1) EP3622066A1 (es)
JP (1) JP7187040B2 (es)
KR (1) KR20200006568A (es)
CN (1) CN110603324A (es)
AU (1) AU2018266879A1 (es)
BR (1) BR112019023800A2 (es)
CA (1) CA3060988A1 (es)
CL (1) CL2019003248A1 (es)
CO (1) CO2019012484A2 (es)
MX (1) MX2019013458A (es)
WO (1) WO2018209192A1 (es)
ZA (1) ZA201907018B (es)

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ES2745287T3 (es) 2013-08-29 2020-02-28 Univ Texas Enzimas degradadoras de cistina/cisteína de primate genomanipuladas como agentes antineogénicos
US20190309271A1 (en) 2018-03-20 2019-10-10 Rubius Therapeutics, Inc. Therapeutic cell systems and methods for treating homocystinuria
CA3136662A1 (en) * 2019-04-10 2020-10-15 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for treating homocystinuria and other conditions
JP2023535537A (ja) * 2020-05-29 2023-08-18 アエグリア バイオセラピューティクス， インク． シスタチオニン－ガンマ－リアーゼを使用するホモシスチン尿症及び高ホモシステイン血症の治療

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KR20200006568A (ko) 2020-01-20
CA3060988A1 (en) 2018-11-15
WO2018209192A8 (en) 2019-01-03
CO2019012484A2 (es) 2020-01-17
EP3622066A1 (en) 2020-03-18
WO2018209192A1 (en) 2018-11-15
AU2018266879A1 (en) 2019-11-07
JP2020519278A (ja) 2020-07-02
ZA201907018B (en) 2022-04-28
BR112019023800A2 (pt) 2020-07-28
MX2019013458A (es) 2020-01-15
JP7187040B2 (ja) 2022-12-12
US20210386838A1 (en) 2021-12-16
US20180326025A1 (en) 2018-11-15
US11033612B2 (en) 2021-06-15
CN110603324A (zh) 2019-12-20

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