MX2019012347A - Optimizacion de terapia de reemplazo enzimatico para el tratamiento de la homocistinuria. - Google Patents

Optimizacion de terapia de reemplazo enzimatico para el tratamiento de la homocistinuria.

Info

Publication number
MX2019012347A
MX2019012347A MX2019012347A MX2019012347A MX2019012347A MX 2019012347 A MX2019012347 A MX 2019012347A MX 2019012347 A MX2019012347 A MX 2019012347A MX 2019012347 A MX2019012347 A MX 2019012347A MX 2019012347 A MX2019012347 A MX 2019012347A
Authority
MX
Mexico
Prior art keywords
htcbs
homocystinuria
treatment
kda
optimization
Prior art date
Application number
MX2019012347A
Other languages
English (en)
Inventor
Majtan Tomas
P Kraus Jan
M Bublil Erez
GLAVIN Frank
Sellos-Moura Marcia
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
Application filed by Univ Colorado Regents filed Critical Univ Colorado Regents
Publication of MX2019012347A publication Critical patent/MX2019012347A/es

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51Lyases (4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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
    • A61K47/59Medicinal 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
    • A61K47/60Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88Lyases (4.)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y402/00Carbon-oxygen lyases (4.2)
    • C12Y402/01Hydro-lyases (4.2.1)
    • C12Y402/01022Cystathionine beta-synthase (4.2.1.22)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente invención proporciona un método de PEGilación de una proteína de cistationina ß-sintasa truncada humana que contiene una mutación de una cisteína a una serina en la posición de aminoácido 15 (htCBS C15S). El htCBS C15S se PEGiló con una de las moléculas de PEG de éster de NHS de 5 kDa, 10 kDa o 20 kDa. La supervisión durante el proceso del proceso de PEGilación se usó en el método para reducir los niveles de htCBS C15S sin PEGilar y htCBS C15S con PEGilación insuficiente. La administración de htCBS C15S PEGilado tuvo eficacia a lo largo del tratamiento para la homocistinuria.
MX2019012347A 2017-04-17 2018-04-17 Optimizacion de terapia de reemplazo enzimatico para el tratamiento de la homocistinuria. MX2019012347A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762486246P 2017-04-17 2017-04-17
PCT/US2018/027854 WO2018195006A1 (en) 2017-04-17 2018-04-17 Optimization of enzyme replacement therapy for treatment of homocystinuria

Publications (1)

Publication Number Publication Date
MX2019012347A true MX2019012347A (es) 2020-01-15

Family

ID=63856394

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019012347A MX2019012347A (es) 2017-04-17 2018-04-17 Optimizacion de terapia de reemplazo enzimatico para el tratamiento de la homocistinuria.

Country Status (8)

Country Link
US (2) US11324811B2 (es)
EP (1) EP3612214A4 (es)
JP (1) JP7146897B2 (es)
CN (2) CN110753556A (es)
AU (1) AU2018254410A1 (es)
CA (1) CA3059592A1 (es)
MX (1) MX2019012347A (es)
WO (1) WO2018195006A1 (es)

Families Citing this family (5)

* 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
SG10201912972SA (en) * 2015-11-09 2020-03-30 Univ Colorado Regents Compositions and methods for treatment of homocystinuria
AU2018254410A1 (en) 2017-04-17 2019-10-10 The Regents Of The University Of Colorado, A Body Corporate Optimization of enzyme replacement therapy for treatment of homocystinuria
JP2022546550A (ja) * 2019-09-03 2022-11-04 トラヴェール セラピューティクス スウィツァランド ゲーエムベーハー 上昇したホモシステインレベルを処置するためのシスタチオニンベータ合成酵素による酵素療法
CN114965761A (zh) * 2022-05-17 2022-08-30 深圳赛保尔生物药业有限公司 聚乙二醇化蛋白药物中n-羟基琥珀酰亚胺的检测方法

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SG10201912972SA (en) * 2015-11-09 2020-03-30 Univ Colorado Regents Compositions and methods for treatment of homocystinuria
AU2018254410A1 (en) 2017-04-17 2019-10-10 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
JP2020516322A (ja) 2020-06-11
US11324811B2 (en) 2022-05-10
CA3059592A1 (en) 2018-10-25
AU2018254410A1 (en) 2019-10-10
US20200261555A1 (en) 2020-08-20
JP7146897B2 (ja) 2022-10-04
CN118064414A (zh) 2024-05-24
EP3612214A4 (en) 2021-01-20
EP3612214A1 (en) 2020-02-26
WO2018195006A1 (en) 2018-10-25
US20230039591A1 (en) 2023-02-09
CN110753556A (zh) 2020-02-04

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