BR112021026292A2 - Cistationina beta sintase peguilada para terapia enzimática para tratamento de homocistinúria - Google Patents

Cistationina beta sintase peguilada para terapia enzimática para tratamento de homocistinúria

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Publication number
BR112021026292A2
BR112021026292A2 BR112021026292A BR112021026292A BR112021026292A2 BR 112021026292 A2 BR112021026292 A2 BR 112021026292A2 BR 112021026292 A BR112021026292 A BR 112021026292A BR 112021026292 A BR112021026292 A BR 112021026292A BR 112021026292 A2 BR112021026292 A2 BR 112021026292A2
Authority
BR
Brazil
Prior art keywords
homocystinuria
treatment
pegylated
enzyme therapy
cystathionine beta
Prior art date
Application number
BR112021026292A
Other languages
English (en)
Inventor
Erez Bublil
Frank Glavin
P Kraus Jan
Marcia Sellos-Moura
Orhan Causevic
Randy Wanner
Tomas Majtan
Original Assignee
Univ Colorado Regents
Travere Therapeutics Switzerland Gmbh
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
Application filed by Univ Colorado Regents, Travere Therapeutics Switzerland Gmbh filed Critical Univ Colorado Regents
Publication of BR112021026292A2 publication Critical patent/BR112021026292A2/pt

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    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K9/14—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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/08—Drugs for disorders of the alimentary tract or the digestive system for nausea, cinetosis or vertigo; Antiemetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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)

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Inorganic Chemistry (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Otolaryngology (AREA)
  • Hematology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Neurosurgery (AREA)
  • Obesity (AREA)
  • Biomedical Technology (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

cistationina beta sintase peguilada para terapia enzimática para tratamento de homocistinúria. a presente invenção refere-se a formulações para um produto farmacêutico que compreende uma proteína cbs peguilada com a sequência de aminoácidos de seq id no: 1. as dosagens e regimes de dosagem são fornecidos para o tratamento da homocistinúria em um indivíduo em necessidade do mesmo. além disso, as dosagens e regimes de dosagem também são fornecidos para reduzir o nível de homocisteína (hcy) ou aumentar os níveis de cisteína (cys) e/ou cistationina (cth) em um indivíduo em necessidade dos mesmos.
BR112021026292A 2019-06-26 2020-06-26 Cistationina beta sintase peguilada para terapia enzimática para tratamento de homocistinúria BR112021026292A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962866810P 2019-06-26 2019-06-26
US202062983860P 2020-03-02 2020-03-02
PCT/US2020/039870 WO2020264333A1 (en) 2019-06-26 2020-06-26 Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria

Publications (1)

Publication Number Publication Date
BR112021026292A2 true BR112021026292A2 (pt) 2022-05-31

Family

ID=71670437

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021026292A BR112021026292A2 (pt) 2019-06-26 2020-06-26 Cistationina beta sintase peguilada para terapia enzimática para tratamento de homocistinúria

Country Status (12)

Country Link
US (1) US20220265835A1 (pt)
EP (1) EP3990005A1 (pt)
JP (1) JP2022539077A (pt)
KR (1) KR20220044726A (pt)
CN (1) CN114786712A (pt)
AU (1) AU2020302079A1 (pt)
BR (1) BR112021026292A2 (pt)
CA (1) CA3145426A1 (pt)
CL (1) CL2021003474A1 (pt)
IL (1) IL289245A (pt)
MX (1) MX2021015966A (pt)
WO (1) WO2020264333A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021046190A1 (en) * 2019-09-03 2021-03-11 Orphan Technologies Ltd. Cystathionine beta-synthase enzyme therapy for treatment of elevated homocysteine levels
CN115721735A (zh) * 2022-10-13 2023-03-03 呈诺再生医学科技(北京)有限公司 Cbs基因在制备糖尿病视网膜病变治疗药物中的应用

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PE20121647A1 (es) * 2009-08-29 2012-12-31 Abbvie Inc Proteinas terapeuticas de union a dll4
EP2510100B1 (en) * 2009-12-09 2017-10-11 CureVac AG Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
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
AU2015241422B2 (en) 2014-04-01 2020-12-03 Rubius Therapeutics, Inc. Methods and compositions for immunomodulation
WO2016183482A1 (en) 2015-05-13 2016-11-17 Rubius Therapeutics, Inc. Membrane-receiver complex therapeutics
LT3373922T (lt) * 2015-11-09 2022-04-11 The Regents Of The University Of Colorado, A Body Corporate Kompozicijos ir būdai skirti naudoti homocistinurijos gydyme
BR112019000195A2 (pt) 2016-07-07 2019-04-24 Rubius Therapeutics, Inc. composições e métodos relacionados a sistemas celulares terapêuticos que expressam rna exógeno

Also Published As

Publication number Publication date
MX2021015966A (es) 2022-04-06
IL289245A (en) 2022-02-01
CN114786712A (zh) 2022-07-22
WO2020264333A1 (en) 2020-12-30
CA3145426A1 (en) 2020-12-30
CL2021003474A1 (es) 2022-09-02
KR20220044726A (ko) 2022-04-11
US20220265835A1 (en) 2022-08-25
AU2020302079A1 (en) 2022-02-17
EP3990005A1 (en) 2022-05-04
JP2022539077A (ja) 2022-09-07

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