CO2019012484A2 - 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
CO2019012484A2
CO2019012484A2 CONC2019/0012484A CO2019012484A CO2019012484A2 CO 2019012484 A2 CO2019012484 A2 CO 2019012484A2 CO 2019012484 A CO2019012484 A CO 2019012484A CO 2019012484 A2 CO2019012484 A2 CO 2019012484A2
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CO
Colombia
Prior art keywords
homocystinuria
decrease
treatment
hyperhomocysteinemia
homocysteine
Prior art date
Application number
CONC2019/0012484A
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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 CO2019012484A2 publication Critical patent/CO2019012484A2/es

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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51—Lyases (4)
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    • 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
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    • 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
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    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
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    • 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
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    • 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]
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    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
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    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
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    • 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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  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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.
CONC2019/0012484A 2017-05-12 2019-11-07 Disminución de homocisteína mediada por enzimas humanas para el tratamiento de pacientes con hiperhomocisteinemia y homocistinuria CO2019012484A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762505493P 2017-05-12 2017-05-12
PCT/US2018/032246 WO2018209192A1 (en) 2017-05-12 2018-05-11 Human-enzyme mediated depletion of homocysteine for treating patients with hyperhomocysteinemia and homocystinuria

Publications (1)

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CO2019012484A2 true CO2019012484A2 (es) 2020-01-17

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CONC2019/0012484A CO2019012484A2 (es) 2017-05-12 2019-11-07 Disminución de homocisteína mediada por enzimas humanas para el tratamiento de pacientes con hiperhomocisteinemia y homocistinuria

Country Status (13)

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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
WO2020210667A1 (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
BR112022024329A2 (pt) * 2020-05-29 2023-01-31 Aeglea Biotherapeutics Inc Tratamento de homocistinúria e hiperhomocisteinemia utilizando cistationina-gama-liase

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WO2015031726A2 (en) * 2013-08-29 2015-03-05 Board Of Regents, The University Of Texas System Engineered primate l-methioninase for therapeutic purposes
ES2745287T3 (es) * 2013-08-29 2020-02-28 Univ Texas Enzimas degradadoras de cistina/cisteína de primate genomanipuladas como agentes antineogénicos
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MX2019013458A (es) 2020-01-15
US20180326025A1 (en) 2018-11-15
CA3060988A1 (en) 2018-11-15
CL2019003248A1 (es) 2020-03-20
US20210386838A1 (en) 2021-12-16
WO2018209192A8 (en) 2019-01-03
EP3622066A1 (en) 2020-03-18
BR112019023800A2 (pt) 2020-07-28
JP2020519278A (ja) 2020-07-02
KR20200006568A (ko) 2020-01-20
CN110603324A (zh) 2019-12-20
ZA201907018B (en) 2022-04-28
JP7187040B2 (ja) 2022-12-12
WO2018209192A1 (en) 2018-11-15
US11033612B2 (en) 2021-06-15
AU2018266879A1 (en) 2019-11-07

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