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
Links
- 206010020365 Homocystinuria Diseases 0.000 title abstract 2
- 238000002641 enzyme replacement therapy Methods 0.000 title 1
- 238000005457 optimization Methods 0.000 title 1
- 102220612388 Mitogen-activated protein kinase kinase kinase 1_C15S_mutation Human genes 0.000 abstract 5
- 238000000034 method Methods 0.000 abstract 3
- 230000006320 pegylation Effects 0.000 abstract 2
- YPWSLBHSMIKTPR-UHFFFAOYSA-N Cystathionine Natural products OC(=O)C(N)CCSSCC(N)C(O)=O YPWSLBHSMIKTPR-UHFFFAOYSA-N 0.000 abstract 1
- ILRYLPWNYFXEMH-UHFFFAOYSA-N D-cystathionine Natural products OC(=O)C(N)CCSCC(N)C(O)=O ILRYLPWNYFXEMH-UHFFFAOYSA-N 0.000 abstract 1
- ILRYLPWNYFXEMH-WHFBIAKZSA-N L-cystathionine Chemical compound [O-]C(=O)[C@@H]([NH3+])CCSC[C@H]([NH3+])C([O-])=O ILRYLPWNYFXEMH-WHFBIAKZSA-N 0.000 abstract 1
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 abstract 1
- 235000001014 amino acid Nutrition 0.000 abstract 1
- 150000001413 amino acids Chemical group 0.000 abstract 1
- 235000018417 cysteine Nutrition 0.000 abstract 1
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 abstract 1
- 150000002148 esters Chemical class 0.000 abstract 1
- 238000010978 in-process monitoring Methods 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
- 235000018102 proteins Nutrition 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
-
- 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
- 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
- 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
-
- 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
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)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Urology & Nephrology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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.
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 (9)
| Country | Link |
|---|---|
| US (2) | US11324811B2 (es) |
| EP (1) | EP3612214A4 (es) |
| JP (1) | JP7146897B2 (es) |
| CN (2) | CN118064414A (es) |
| AU (1) | AU2018254410B2 (es) |
| CA (1) | CA3059592A1 (es) |
| IL (1) | IL269544B2 (es) |
| MX (1) | MX2019012347A (es) |
| WO (1) | WO2018195006A1 (es) |
Families Citing this family (6)
| 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 |
| BR112018007768A2 (pt) * | 2015-11-09 | 2018-10-23 | Univ Colorado Regents | composições e métodos para tratamento de homocistinúria |
| JP7146897B2 (ja) | 2017-04-17 | 2022-10-04 | ザ リージェンツ オブ ザ ユニバーシティ オブ コロラド，ア ボディー コーポレイト | ホモシスチン尿症の治療のための酵素補充療法の最適化 |
| DK3990005T3 (da) | 2019-06-26 | 2025-12-08 | Travere Therapeutics Switzerland Gmbh | Pegyleret cystathioninbetasynthase til enzymterapi til behandling af homocystinuri |
| EP4025243A4 (en) * | 2019-09-03 | 2023-09-27 | Travere Therapeutics Switzerland GmbH | CYSTATHIONINE BETA-SYNTHASE ENZYMA THERAPY FOR THE TREATMENT OF HIGH HOMOCYSTEINE LEVELS |
| CN114965761B (zh) * | 2022-05-17 | 2024-07-02 | 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 |
Family Cites Families (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS5576559A (en) | 1978-12-01 | 1980-06-09 | Chiyou Lsi Gijutsu Kenkyu Kumiai | Electron beam fine adjusting device |
| JPS57209206A (en) | 1981-06-08 | 1982-12-22 | Kanebo Ltd | Skin type pack agent |
| 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 |
| US5643575A (en) | 1993-10-27 | 1997-07-01 | Enzon, Inc. | Non-antigenic branched polymer conjugates |
| US5919455A (en) | 1993-10-27 | 1999-07-06 | 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 |
| EP1221615A2 (en) | 1995-04-25 | 2002-07-10 | Oridigm Corporation | S-adenosyl methionine regulation of metabolic pathways and its use in 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 |
| CN100335503C (zh) * | 1998-04-28 | 2007-09-05 | 应用研究系统Ars股份公司 | 多元醇干扰素β偶联物 |
| US6174696B1 (en) | 1999-11-12 | 2001-01-16 | Genzyme Corporation | Method for the determination of homocysteine |
| WO2001002600A2 (en) | 1999-07-06 | 2001-01-11 | General Atomics | Detection of analytes using attenuated enzymes |
| US20030091543A1 (en) | 2001-10-26 | 2003-05-15 | Klein Matthew B. | Therapeutic cell preparation grafts and methods of use thereof |
| US7485307B2 (en) | 2002-06-17 | 2009-02-03 | The Regents Of The University Of Colorado | Human cystathionine β-synthase variants and methods of production 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 |
| US20050036981A1 (en) | 2003-07-31 | 2005-02-17 | Shigeo Yagi | USE OF PLP WITH PEG-rMETase IN VIVO FOR ENHANCED EFFICACY |
| 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 |
| US8500720B2 (en) | 2005-05-09 | 2013-08-06 | Medtronic, Inc | Method and apparatus for treatment of cardiac disorders |
| US20070010492A1 (en) | 2005-07-11 | 2007-01-11 | Generale Robert J | Composition and method for reducing homocysteine caused by drugs containing methyl compounds |
| EP1878739A1 (en) | 2006-07-14 | 2008-01-16 | LEK Pharmaceuticals D.D. | One step IMAC (MCAC) purification of proteins |
| 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 |
| CN101322840A (zh) | 2008-05-28 | 2008-12-17 | 中国人民解放军军事医学科学院卫生学环境医学研究所 | 一种人胱硫醚β-合成酶重组蛋白及应用 |
| JP2011084559A (ja) * | 2009-09-16 | 2011-04-28 | Institute Of Physical & Chemical Research | 改変融合タンパク質 |
| WO2011097381A2 (en) | 2010-02-04 | 2011-08-11 | The Board Of Regents Of Use University Of Texas System | Engineered enzymes with methionine-gamma-lyase enzymes and pharmacological preparations therof |
| CA2803668A1 (en) | 2010-07-01 | 2012-01-05 | 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 |
| EP3325496B1 (en) * | 2015-07-24 | 2024-02-07 | Hanmi Pharm. Co., Ltd. | Method of preparing physiologically active polypeptide conjugate |
| BR112018007768A2 (pt) | 2015-11-09 | 2018-10-23 | Univ Colorado Regents | composições e métodos para tratamento de homocistinúria |
| JP7146897B2 (ja) | 2017-04-17 | 2022-10-04 | ザ リージェンツ オブ ザ ユニバーシティ オブ コロラド，ア ボディー コーポレイト | ホモシスチン尿症の治療のための酵素補充療法の最適化 |
| DK3990005T3 (da) | 2019-06-26 | 2025-12-08 | Travere Therapeutics Switzerland Gmbh | Pegyleret cystathioninbetasynthase til enzymterapi til behandling af homocystinuri |
| EP4025243A4 (en) | 2019-09-03 | 2023-09-27 | Travere Therapeutics Switzerland GmbH | CYSTATHIONINE BETA-SYNTHASE ENZYMA THERAPY FOR THE TREATMENT OF HIGH HOMOCYSTEINE LEVELS |
-
2018
- 2018-04-17 JP JP2020506311A patent/JP7146897B2/ja active Active
- 2018-04-17 EP EP18787619.8A patent/EP3612214A4/en active Pending
- 2018-04-17 CN CN202410188237.0A patent/CN118064414A/zh active Pending
- 2018-04-17 AU AU2018254410A patent/AU2018254410B2/en active Active
- 2018-04-17 CN CN201880025544.XA patent/CN110753556A/zh active Pending
- 2018-04-17 WO PCT/US2018/027854 patent/WO2018195006A1/en not_active Ceased
- 2018-04-17 MX MX2019012347A patent/MX2019012347A/es unknown
- 2018-04-17 IL IL269544A patent/IL269544B2/en unknown
- 2018-04-17 CA CA3059592A patent/CA3059592A1/en active Pending
- 2018-04-17 US US16/605,918 patent/US11324811B2/en active Active
-
2022
- 2022-04-05 US US17/713,891 patent/US12042529B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| AU2018254410B2 (en) | 2025-02-06 |
| EP3612214A4 (en) | 2021-01-20 |
| IL269544A (en) | 2019-11-28 |
| JP2020516322A (ja) | 2020-06-11 |
| AU2018254410A1 (en) | 2019-10-10 |
| IL269544B1 (en) | 2024-09-01 |
| CN118064414A (zh) | 2024-05-24 |
| US11324811B2 (en) | 2022-05-10 |
| US20230039591A1 (en) | 2023-02-09 |
| US20200261555A1 (en) | 2020-08-20 |
| IL269544B2 (en) | 2025-01-01 |
| JP7146897B2 (ja) | 2022-10-04 |
| WO2018195006A1 (en) | 2018-10-25 |
| EP3612214A1 (en) | 2020-02-26 |
| CA3059592A1 (en) | 2018-10-25 |
| CN110753556A (zh) | 2020-02-04 |
| US12042529B2 (en) | 2024-07-23 |
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