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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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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
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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
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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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    • C12N15/867—Retroviral vectors
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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.)
    • 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
    • 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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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
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  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
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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)
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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ID=62685099

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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

Country Status (14)

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

Families Citing this family (5)

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KR102269209B1 (ko) 2013-08-29 2021-06-28 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 항신생물제로서 조작된 영장류 시스틴/시스테인 분해 효소
US20190309271A1 (en) 2018-03-20 2019-10-10 Rubius Therapeutics, Inc. Therapeutic cell systems and methods for treating homocystinuria
SG11202104205XA (en) 2018-10-26 2021-05-28 Univ Texas Engineered primate cystine/cysteine degrading enzymes for therapeutic uses
AU2020272040C1 (en) * 2019-04-10 2024-03-28 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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WO2004112717A2 (en) 2003-06-17 2004-12-29 Harrison Roger G Conjugate for the specific targeting of anticancer agents to cancer cells and production thereof
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KR20060065663A (ko) 2003-07-31 2006-06-14 안티캔서, 인코포레이티드 생체 내에서의 유효성 향상을 위한, ＰＥＧ-ｒＭＥＴ아제와조합된 ＰＬＰ의 용도
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US9034318B2 (en) 2011-08-30 2015-05-19 The Regents Of The University Of Colorado, A Body Corporate Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
KR102269209B1 (ko) 2013-08-29 2021-06-28 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 항신생물제로서 조작된 영장류 시스틴/시스테인 분해 효소
KR102244750B1 (ko) 2013-08-29 2021-04-28 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 치료적 목적을 위한 조작된 영장류 ｌ-메티오니나제
CA3028771A1 (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

Also Published As

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

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