CA3060988A1 - Human-enzyme mediated depletion of homocysteine for treating patients with hyperhomocysteinemia and homocystinuria - Google Patents

Human-enzyme mediated depletion of homocysteine for treating patients with hyperhomocysteinemia and homocystinuria Download PDF

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Publication number
CA3060988A1
CA3060988A1 CA3060988A CA3060988A CA3060988A1 CA 3060988 A1 CA3060988 A1 CA 3060988A1 CA 3060988 A CA3060988 A CA 3060988A CA 3060988 A CA3060988 A CA 3060988A CA 3060988 A1 CA3060988 A1 CA 3060988A1
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Prior art keywords
enzyme
cgl
leucine
modified
valine
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CA3060988A
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English (en)
French (fr)
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George Georgiou
Everett Stone
Wei-Cheng Lu
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University of Texas System
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University of Texas System
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    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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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    • C12N15/63—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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    • 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
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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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
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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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  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (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)
CA3060988A 2017-05-12 2018-05-11 Human-enzyme mediated depletion of homocysteine for treating patients with hyperhomocysteinemia and homocystinuria Pending CA3060988A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762505493P 2017-05-12 2017-05-12
US62/505,493 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)

Publication Number Publication Date
CA3060988A1 true CA3060988A1 (en) 2018-11-15

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CA3060988A Pending CA3060988A1 (en) 2017-05-12 2018-05-11 Human-enzyme mediated depletion of homocysteine for treating patients with hyperhomocysteinemia and homocystinuria

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US (2) US11033612B2 (enExample)
EP (1) EP3622066A1 (enExample)
JP (1) JP7187040B2 (enExample)
KR (1) KR102680776B1 (enExample)
CN (1) CN110603324A (enExample)
AU (1) AU2018266879B2 (enExample)
BR (1) BR112019023800A2 (enExample)
CA (1) CA3060988A1 (enExample)
CL (1) CL2019003248A1 (enExample)
CO (1) CO2019012484A2 (enExample)
IL (1) IL270503B2 (enExample)
MX (1) MX386601B (enExample)
WO (1) WO2018209192A1 (enExample)
ZA (1) ZA201907018B (enExample)

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Publication number Priority date Publication date Assignee Title
US10363311B2 (en) 2013-08-29 2019-07-30 Board Of Regents, The University Of Texas System Engineered primate cystine/cysteine degrading enzymes as antineogenic agents
US20190309271A1 (en) 2018-03-20 2019-10-10 Rubius Therapeutics, Inc. Therapeutic cell systems and methods for treating homocystinuria
AR116866A1 (es) * 2018-10-26 2021-06-23 Univ Texas Enzimas de degradación de cistina / cisteína de primates modificadas por ingeniería genética
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
WO2021242258A1 (en) * 2020-05-29 2021-12-02 Aeglea Biotherapeutics, Inc. Treatment of homocystinuria and hyperhomocysteinemia using cystathionine-gamma-lyase

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JP6572212B2 (ja) 2013-08-29 2019-09-04 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム 治療用の改変霊長類ｌ−メチオニナーゼ
US10363311B2 (en) 2013-08-29 2019-07-30 Board Of Regents, The University Of Texas System Engineered primate cystine/cysteine degrading enzymes as antineogenic agents
US20180008681A1 (en) 2016-07-06 2018-01-11 Board Of Regents, The University Of Texas System Human-enzyme mediated depletion of cystine for treating patients with cystinuria

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BR112019023800A2 (pt) 2020-07-28
KR102680776B1 (ko) 2024-07-04
IL270503B1 (en) 2024-06-01
US12036269B2 (en) 2024-07-16
EP3622066A1 (en) 2020-03-18
CN110603324A (zh) 2019-12-20
ZA201907018B (en) 2022-04-28
US20180326025A1 (en) 2018-11-15
CL2019003248A1 (es) 2020-03-20
WO2018209192A1 (en) 2018-11-15
US11033612B2 (en) 2021-06-15
JP2020519278A (ja) 2020-07-02
AU2018266879B2 (en) 2024-09-05
MX2019013458A (es) 2020-01-15
US20210386838A1 (en) 2021-12-16
JP7187040B2 (ja) 2022-12-12
KR20200006568A (ko) 2020-01-20
IL270503B2 (en) 2024-10-01
AU2018266879A1 (en) 2019-11-07
MX386601B (es) 2025-03-19
WO2018209192A8 (en) 2019-01-03
IL270503A (enExample) 2020-01-30
CO2019012484A2 (es) 2020-01-17

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