JP2022539077A - ホモシスチン尿症を処置するための酵素療法のためのｐｅｇ化シスタチオニンベータ合成酵素 - Google Patents

ホモシスチン尿症を処置するための酵素療法のためのｐｅｇ化シスタチオニンベータ合成酵素 Download PDF

Info

Publication number
JP2022539077A
JP2022539077A JP2021576921A JP2021576921A JP2022539077A JP 2022539077 A JP2022539077 A JP 2022539077A JP 2021576921 A JP2021576921 A JP 2021576921A JP 2021576921 A JP2021576921 A JP 2021576921A JP 2022539077 A JP2022539077 A JP 2022539077A
Authority
JP
Japan
Prior art keywords
patients
hcy
levels
incorporated
entirety
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2021576921A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022539077A5 (cg-RX-API-DMAC10.html
Inventor
ピー． クラウス、ジャン
ヴァナー、ランディ
ツァウセヴィッチ、オルハン
バブリル、エレズ
グラビン、フランク
セロス－モウラ、マルシア
マイタン、トーマス
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Travere Therapeutics Switzerland GmbH
University of Colorado Colorado Springs
Original Assignee
Travere Therapeutics Switzerland GmbH
University of Colorado Colorado Springs
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 Travere Therapeutics Switzerland GmbH, University of Colorado Colorado Springs filed Critical Travere Therapeutics Switzerland GmbH
Publication of JP2022539077A publication Critical patent/JP2022539077A/ja
Publication of JP2022539077A5 publication Critical patent/JP2022539077A5/ja
Priority to JP2025093526A priority Critical patent/JP2025143275A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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)

Landscapes

  • 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Inorganic Chemistry (AREA)
  • Hospice & Palliative Care (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biomedical Technology (AREA)
  • Otolaryngology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2021576921A 2019-06-26 2020-06-26 ホモシスチン尿症を処置するための酵素療法のためのｐｅｇ化シスタチオニンベータ合成酵素 Pending JP2022539077A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025093526A JP2025143275A (ja) 2019-06-26 2025-06-04 ホモシスチン尿症を処置するための酵素療法のためのｐｅｇ化シスタチオニンベータ合成酵素

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962866810P 2019-06-26 2019-06-26
US62/866,810 2019-06-26
US202062983860P 2020-03-02 2020-03-02
US62/983,860 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025093526A Division JP2025143275A (ja) 2019-06-26 2025-06-04 ホモシスチン尿症を処置するための酵素療法のためのｐｅｇ化シスタチオニンベータ合成酵素

Publications (2)

Publication Number Publication Date
JP2022539077A true JP2022539077A (ja) 2022-09-07
JP2022539077A5 JP2022539077A5 (cg-RX-API-DMAC10.html) 2023-07-03

Family

ID=71670437

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021576921A Pending JP2022539077A (ja) 2019-06-26 2020-06-26 ホモシスチン尿症を処置するための酵素療法のためのｐｅｇ化シスタチオニンベータ合成酵素
JP2025093526A Pending JP2025143275A (ja) 2019-06-26 2025-06-04 ホモシスチン尿症を処置するための酵素療法のためのｐｅｇ化シスタチオニンベータ合成酵素

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025093526A Pending JP2025143275A (ja) 2019-06-26 2025-06-04 ホモシスチン尿症を処置するための酵素療法のためのｐｅｇ化シスタチオニンベータ合成酵素

Country Status (24)

Country Link
US (1) US12364765B2 (cg-RX-API-DMAC10.html)
EP (2) EP3990005B1 (cg-RX-API-DMAC10.html)
JP (2) JP2022539077A (cg-RX-API-DMAC10.html)
KR (1) KR20220044726A (cg-RX-API-DMAC10.html)
CN (1) CN114786712A (cg-RX-API-DMAC10.html)
AU (1) AU2020302079A1 (cg-RX-API-DMAC10.html)
BR (1) BR112021026292A2 (cg-RX-API-DMAC10.html)
CA (1) CA3145426A1 (cg-RX-API-DMAC10.html)
CL (1) CL2021003474A1 (cg-RX-API-DMAC10.html)
DK (1) DK3990005T3 (cg-RX-API-DMAC10.html)
ES (1) ES3058596T3 (cg-RX-API-DMAC10.html)
FI (1) FI3990005T3 (cg-RX-API-DMAC10.html)
HR (1) HRP20251579T1 (cg-RX-API-DMAC10.html)
IL (1) IL289245B2 (cg-RX-API-DMAC10.html)
LT (1) LT3990005T (cg-RX-API-DMAC10.html)
MX (2) MX2021015966A (cg-RX-API-DMAC10.html)
PH (1) PH12021553235A1 (cg-RX-API-DMAC10.html)
PL (1) PL3990005T3 (cg-RX-API-DMAC10.html)
PT (1) PT3990005T (cg-RX-API-DMAC10.html)
RS (1) RS67521B1 (cg-RX-API-DMAC10.html)
SA (1) SA521431197B1 (cg-RX-API-DMAC10.html)
SI (1) SI3990005T1 (cg-RX-API-DMAC10.html)
SM (1) SMT202600042T1 (cg-RX-API-DMAC10.html)
WO (1) WO2020264333A1 (cg-RX-API-DMAC10.html)

Families Citing this family (8)

* 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
DK3998067T3 (da) 2015-11-09 2024-10-28 Univ Colorado Regents Sammensætninger og fremgangsmåder til behandling af homocystinuri
CA3059592A1 (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
MX2021015966A (es) 2019-06-26 2022-04-06 Travere Therapeutics Switzerland Gmbh Producto farmacológico para terapia enzimática para el tratamiento de la homocistinuria.
EP4025243A4 (en) * 2019-09-03 2023-09-27 Travere Therapeutics Switzerland GmbH CYSTATHIONINE BETA-SYNTHASE ENZYMA THERAPY FOR THE TREATMENT OF HIGH 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
CN115671059B (zh) * 2022-11-17 2024-06-11 兆科药业（合肥）有限公司 一种盐酸氨酮戊酸冻干制剂及制备方法
CN120610012A (zh) * 2023-10-20 2025-09-09 T&D诊断加拿大私人有限公司 用于诊断个体整体健康状况的标志物以及装置

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004505024A (ja) * 2000-07-26 2004-02-19 ウイスコンシン　アラムニ　リサーチ　ファンデーション 生物学的物質用の保存及び貯蔵媒体
JP2016506727A (ja) * 2013-01-29 2016-03-07 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ＴＨＥ ＲＥＧＥＮＴＳ ＯＦ ＴＨＥ ＵＮＩＶＥＲＳＩＴＹ ＯＦ ＣＯＬＯＲＡＤＯ，ａ ｂｏｄｙ ｃｏｒｐｏｒａｔｅ ホモシスチン尿症を処置するためのシスタチオニンβ−シンターゼ酵素

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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
EP1396537A1 (en) 2002-09-04 2004-03-10 Avidis SA Treatment of elevated plasma homocysteine
CA2534995A1 (en) 2003-07-31 2005-05-19 Anticancer, Inc. The use of plp with peg-rmetase in vivo for enhanced efficacy
US20070010492A1 (en) 2005-07-11 2007-01-11 Generale Robert J Composition and method for reducing homocysteine caused by drugs containing methyl compounds
CA2771575A1 (en) 2009-08-29 2011-03-03 Abbott Laboratories Therapeutic dll4 binding proteins
EP2510100B1 (en) * 2009-12-09 2017-10-11 CureVac AG Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
BR112013000027A2 (pt) 2010-07-01 2017-07-11 Isis Innovation tratamento de distúrbios cognitivos
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
BR112016011195A2 (pt) 2013-11-18 2017-09-19 Rubius Therapeutics Inc Células eritroides enucleadas e seus métodos de fabricação, composição farmacêutica e seu uso, uso de uma população de células eritroides, biorreator, mistura de células e dispositivo médico
CN106456744A (zh) 2014-04-01 2017-02-22 鲁比厄斯治疗法股份有限公司 用于免疫调节的方法和组合物
WO2016183482A1 (en) 2015-05-13 2016-11-17 Rubius Therapeutics, Inc. Membrane-receiver complex therapeutics
DK3998067T3 (da) * 2015-11-09 2024-10-28 Univ Colorado Regents Sammensætninger og fremgangsmåder til behandling af homocystinuri
MX2019000205A (es) 2016-07-07 2019-09-23 Rubius Therapeutics Inc Composiciones y metodos relacionados con sistemas celulares terapeuticos que expresan arn exogeno.
CA3059592A1 (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
MX2021015966A (es) 2019-06-26 2022-04-06 Travere Therapeutics Switzerland Gmbh Producto farmacológico para terapia enzimática para el tratamiento de la homocistinuria.
EP4025243A4 (en) * 2019-09-03 2023-09-27 Travere Therapeutics Switzerland GmbH CYSTATHIONINE BETA-SYNTHASE ENZYMA THERAPY FOR THE TREATMENT OF HIGH HOMOCYSTEINE LEVELS

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004505024A (ja) * 2000-07-26 2004-02-19 ウイスコンシン　アラムニ　リサーチ　ファンデーション 生物学的物質用の保存及び貯蔵媒体
JP2016506727A (ja) * 2013-01-29 2016-03-07 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ＴＨＥ ＲＥＧＥＮＴＳ ＯＦ ＴＨＥ ＵＮＩＶＥＲＳＩＴＹ ＯＦ ＣＯＬＯＲＡＤＯ，ａ ｂｏｄｙ ｃｏｒｐｏｒａｔｅ ホモシスチン尿症を処置するためのシスタチオニンβ−シンターゼ酵素

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
伊豆津 健一: "タンパク質医薬品の凍結乾燥", 薬剤学, vol. 72巻, 6号, JPN6024013870, 2012, pages 353 - 358, ISSN: 0005674799 *
伊豆津 健一: "凍結溶液の熱測定と凍結乾燥製剤の製剤設計", 熱測定, vol. 36巻, 2号, JPN6024013871, 2009, pages 112 - 120, ISSN: 0005674798 *

Also Published As

Publication number Publication date
US12364765B2 (en) 2025-07-22
EP3990005B1 (en) 2025-08-27
MX2026001319A (es) 2026-03-02
ES3058596T3 (en) 2026-03-11
EP3990005A1 (en) 2022-05-04
BR112021026292A2 (pt) 2022-05-31
US20220265835A1 (en) 2022-08-25
JP2025143275A (ja) 2025-10-01
HRP20251579T1 (hr) 2026-01-30
FI3990005T3 (fi) 2025-11-28
KR20220044726A (ko) 2022-04-11
CA3145426A1 (en) 2020-12-30
PL3990005T4 (pl) 2026-03-02
SI3990005T1 (sl) 2026-02-27
SMT202600042T1 (it) 2026-03-09
WO2020264333A1 (en) 2020-12-30
DK3990005T3 (da) 2025-12-08
SA521431197B1 (ar) 2024-12-03
IL289245A (en) 2022-02-01
PL3990005T3 (pl) 2026-03-02
MX2021015966A (es) 2022-04-06
LT3990005T (lt) 2026-01-12
PH12021553235A1 (en) 2022-08-22
EP4663239A2 (en) 2025-12-17
AU2020302079A1 (en) 2022-02-17
IL289245B2 (en) 2025-09-01
PT3990005T (pt) 2025-12-17
IL289245B1 (en) 2025-05-01
CL2021003474A1 (es) 2022-09-02
EP4663239A3 (en) 2026-03-11
CN114786712A (zh) 2022-07-22
RS67521B1 (sr) 2026-01-30

Similar Documents

Publication Publication Date Title
US12364765B2 (en) Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria
TWI547278B (zh) 阿茲海默症及老化腦部之補缺（ａｎａｐｌｅｒｏｔｉｃ）療法
JP2025183204A (ja) 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
Puddey et al. Regular alcohol use raises blood pressure in treated hypertensive subjects: a randomised controlled trial
ES2668079T3 (es) Composiciones y métodos para tratar la enfermedad de Gaucher
Gottlieb et al. Cardioprotection requires taking out the trash
JP2025016455A (ja) 組換えヒト酸性アルファグリコシダーゼ
BR122019000505B1 (pt) Fosfatase alcalina direcionada ao osso, kits e métodos de seu uso
US20140213559A1 (en) Compositions and treatments for dystrophies
JP2022050589A (ja) ホモシスチン尿症の治療する組成物および方法
US20210371839A1 (en) Products and Methods for Assessing and Increasing Klotho Protein Levels
TW201919689A (zh) 酸性神經髓磷脂酶缺乏症病患異常骨狀況之治療
Ziegler et al. Inborn errors of amino acid metabolism–from underlying pathophysiology to therapeutic advances
JP2025061057A (ja) アルカリホスファターゼポリペプチド及びその使用方法
KR20250078929A (ko) 알츠하이머병 치료를 위한 면역치료법과 사일로이노시톨 병용
RU2839909C2 (ru) Пэгилированная цистатионин-бета-синтаза для ферментотерапии для лечения гомоцистинурии
HK40071277B (en) Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria
HK40071277A (en) Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria
RU2833879C1 (ru) Терапия с использованием фермента цистатионин-бета-синтазы для лечения заболеваний, вызываемых повышенными уровнями гомоцистеина
Herrmann et al. World Congress on Hyperhomocysteinemia
EA045409B1 (ru) Рекомбинантная человеческая кислая альфа-глюкозидаза
BR122024007742A2 (pt) Uso de fosfatase alcalina recombinante no tratamento de ataques
US20190201554A1 (en) Use of lyso-gb1 as druggable target

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230623

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230623

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240416

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240716

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20250204

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250604