BR112018007768A2 - composições e métodos para tratamento de homocistinúria - Google Patents

composições e métodos para tratamento de homocistinúria

Info

Publication number
BR112018007768A2
BR112018007768A2 BR112018007768A BR112018007768A BR112018007768A2 BR 112018007768 A2 BR112018007768 A2 BR 112018007768A2 BR 112018007768 A BR112018007768 A BR 112018007768A BR 112018007768 A BR112018007768 A BR 112018007768A BR 112018007768 A2 BR112018007768 A2 BR 112018007768A2
Authority
BR
Brazil
Prior art keywords
compositions
methods
homocystinuria
treating
treating homocystinuria
Prior art date
Application number
BR112018007768A
Other languages
English (en)
Inventor
Bublil Erez
P Kraus Jan
Majtan Tomas
Original Assignee
Univ Colorado Regents
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from US14/935,690 external-priority patent/US9675678B2/en
Application filed by Univ Colorado Regents filed Critical Univ Colorado Regents
Publication of BR112018007768A2 publication Critical patent/BR112018007768A2/pt

Links

Classifications

    • 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
    • A61K31/185—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, pantothenic acid
    • A61K31/198—Alpha-aminoacids, e.g. alanine, edetic acids [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
    • A61K31/38—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

Abstract

a presente invenção refere-se a composições e métodos para a terapia de reposição enzimática usando cistationina beta sintase humana modificada (cbs) no tratamento de homocistinúria e doenças e distúrbios relacionados.
BR112018007768A 2015-11-09 2016-11-09 composições e métodos para tratamento de homocistinúria BR112018007768A2 (pt)

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
BR112018007768A2 true BR112018007768A2 (pt) 2018-10-23

Family

ID=58696136

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018007768A BR112018007768A2 (pt) 2015-11-09 2016-11-09 composições e métodos para tratamento de homocistinúria

Country Status (24)

Country Link
US (3) US11077175B2 (pt)
EP (2) EP3998067A1 (pt)
JP (1) JP7009364B2 (pt)
KR (1) KR20180074703A (pt)
CN (2) CN108472277A (pt)
AU (2) AU2016354030B2 (pt)
BR (1) BR112018007768A2 (pt)
CA (1) CA3001625A1 (pt)
DK (1) DK3373922T3 (pt)
ES (1) ES2909914T3 (pt)
HK (1) HK1258502A1 (pt)
HR (1) HRP20220407T1 (pt)
HU (1) HUE058749T2 (pt)
IL (2) IL302198A (pt)
LT (1) LT3373922T (pt)
MX (1) MX2018005312A (pt)
NZ (1) NZ741552A (pt)
PL (1) PL3373922T3 (pt)
PT (1) PT3373922T (pt)
RS (1) RS63070B1 (pt)
SG (1) SG10201912972SA (pt)
SI (1) SI3373922T1 (pt)
WO (1) WO2017083327A1 (pt)
ZA (1) ZA201802376B (pt)

Families Citing this family (4)

* 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
IL302198A (en) 2015-11-09 2023-06-01 Univ Colorado Regents Preparations and methods for the treatment of excessive homocysteine in the urine
WO2018195006A1 (en) 2017-04-17 2018-10-25 The Regents Of The University Of Colorado, A Body Corporate Optimization of enzyme replacement therapy for treatment of homocystinuria
US20220265835A1 (en) 2019-06-26 2022-08-25 The Regents Of The University Of Colorado, A Body Corporate Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria

Family Cites Families (30)

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JPS5576559A (en) 1978-12-01 1980-06-09 Chiyou Lsi Gijutsu Kenkyu Kumiai Electron beam fine adjusting device
US5656425A (en) 1993-08-17 1997-08-12 Regents Of The University Of Colorado Screening for mutations by expressing cDNA segments
US5523225A (en) 1993-09-13 1996-06-04 Regents Of The University Of Colorado DNA sequence encoding human cystathionine β-synthase
US5919455A (en) 1993-10-27 1999-07-06 Enzon, Inc. Non-antigenic branched polymer conjugates
US5643575A (en) 1993-10-27 1997-07-01 Enzon, Inc. Non-antigenic branched polymer conjugates
US5730990A (en) 1994-06-24 1998-03-24 Enzon, Inc. Non-antigenic amine derived polymers and polymer conjugates
US5635375A (en) 1995-01-09 1997-06-03 Regents Of The University Of Colorado Method of increasing the yield and heme saturation of cystathione β-synthase
EP0824345A4 (en) 1995-04-25 1999-08-25 Oridigm Corp REGULATION OF METABOLIC S-ADENOSYL METHIONINE AND APPLICATION TO DIAGNOSIS AND THERAPY
US5705151A (en) 1995-05-18 1998-01-06 National Jewish Center For Immunology & Respiratory Medicine Gene therapy for T cell regulation
US6153655A (en) 1998-04-17 2000-11-28 Enzon, Inc. Terminally-branched polymeric linkers and polymeric conjugates containing the same
US6174696B1 (en) 1999-11-12 2001-01-16 Genzyme Corporation Method for the determination of homocysteine
AU5781800A (en) 1999-07-06 2001-01-22 General Atomics Methods and compositions for assaying analytes
US20030091543A1 (en) 2001-10-26 2003-05-15 Klein Matthew B. Therapeutic cell preparation grafts and methods of use thereof
WO2003106971A2 (en) 2002-06-17 2003-12-24 The Regents Of The University Of Colorado HUMAN CYSTATHIONINE β-SYNTHASE VARIANTS AND METHODS OF PRODU CTION THEREOF
GB0216001D0 (en) 2002-07-10 2002-08-21 Nat Blood Authority Process and composition
EP1396537A1 (en) * 2002-09-04 2004-03-10 Avidis SA Treatment of elevated plasma homocysteine
CN1552905A (zh) 2003-12-18 2004-12-08 中国农业科学院茶叶研究所 一类茶树胱硫醚-γ-合成酶特异表达序列标签及其生物芯片
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JP5074146B2 (ja) 2007-03-30 2012-11-14 オリンパス株式会社 カプセル型医療装置
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AU2010356582A1 (en) 2010-07-01 2013-02-21 Isis Innovation Limited Treatment of cognitive disorders
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
US9243239B2 (en) * 2012-03-26 2016-01-26 The Regents Of The University Of Colorado, A Body Corporate Purification of cystathionine beta-synthase
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
WO2015033279A1 (en) * 2013-09-04 2015-03-12 Mahesh Kandula Compositions and methods for the treatment of homocystinuria
IL302198A (en) 2015-11-09 2023-06-01 Univ Colorado Regents Preparations and methods for the treatment of excessive homocysteine in the urine
WO2018195006A1 (en) * 2017-04-17 2018-10-25 The Regents Of The University Of Colorado, A Body Corporate Optimization of enzyme replacement therapy for treatment of homocystinuria

Also Published As

Publication number Publication date
HRP20220407T1 (hr) 2022-05-27
PT3373922T (pt) 2022-03-28
AU2016354030B2 (en) 2022-09-15
ES2909914T3 (es) 2022-05-10
AU2016354030A1 (en) 2018-05-10
AU2022287540A1 (en) 2023-02-02
WO2017083327A1 (en) 2017-05-18
MX2018005312A (es) 2018-08-15
SI3373922T1 (sl) 2022-05-31
US20240091325A1 (en) 2024-03-21
IL302198A (en) 2023-06-01
US20200276282A1 (en) 2020-09-03
CN108472277A (zh) 2018-08-31
CA3001625A1 (en) 2017-05-18
HUE058749T2 (hu) 2022-09-28
EP3373922B1 (en) 2022-01-05
IL258487A (en) 2018-05-31
EP3373922A1 (en) 2018-09-19
DK3373922T3 (da) 2022-03-28
IL258487B2 (en) 2023-09-01
HK1258502A1 (zh) 2019-11-15
US20220125896A1 (en) 2022-04-28
KR20180074703A (ko) 2018-07-03
US11077175B2 (en) 2021-08-03
CN115920016A (zh) 2023-04-07
IL258487B1 (en) 2023-05-01
RS63070B1 (sr) 2022-04-29
SG10201912972SA (en) 2020-03-30
NZ741552A (en) 2022-04-29
JP2018532763A (ja) 2018-11-08
JP7009364B2 (ja) 2022-02-10
JP2022050589A (ja) 2022-03-30
PL3373922T3 (pl) 2022-05-02
US11771745B2 (en) 2023-10-03
EP3998067A1 (en) 2022-05-18
LT3373922T (lt) 2022-04-11
ZA201802376B (en) 2020-11-25
EP3373922A4 (en) 2019-05-15

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