KR20220044726A - 호모시스틴요증 치료용 효소 치료요법을 위한 페길화된 시스타티오닌 베타 신타제 - Google Patents

호모시스틴요증 치료용 효소 치료요법을 위한 페길화된 시스타티오닌 베타 신타제 Download PDF

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KR20220044726A
KR20220044726A KR1020227002804A KR20227002804A KR20220044726A KR 20220044726 A KR20220044726 A KR 20220044726A KR 1020227002804 A KR1020227002804 A KR 1020227002804A KR 20227002804 A KR20227002804 A KR 20227002804A KR 20220044726 A KR20220044726 A KR 20220044726A
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Inventor
에레즈 버블릴
프랭크 글래빈
마르시아 셀로스-모라
토마스 마잔
얀 피. 크라우스
랜디 워너
오르한 카우세빅
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트래비어 써래페틱스 스위처랜드 게엠베하
더 리젠츠 오브 더 유니버시티 오브 콜로라도, 어 바디 코퍼레이트
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Publication of KR20220044726A publication Critical patent/KR20220044726A/ko
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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)
  • Veterinary Medicine (AREA)
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  • 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)
KR1020227002804A 2019-06-26 2020-06-26 호모시스틴요증 치료용 효소 치료요법을 위한 페길화된 시스타티오닌 베타 신타제 Pending KR20220044726A (ko)

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

Publications (1)

Publication Number Publication Date
KR20220044726A true KR20220044726A (ko) 2022-04-11

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ID=71670437

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Application Number Title Priority Date Filing Date
KR1020227002804A Pending KR20220044726A (ko) 2019-06-26 2020-06-26 호모시스틴요증 치료용 효소 치료요법을 위한 페길화된 시스타티오닌 베타 신타제

Country Status (24)

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

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3373922B1 (en) 2015-11-09 2022-01-05 The Regents of The University of Colorado, A Body Corporate Compositions and methods for use in the treatment of homocystinuria
EP3612214A4 (en) 2017-04-17 2021-01-20 The Regents of the University of Colorado, A Body Corporate ENZYME REPLACEMENT THERAPY OPTIMIZATION FOR THE TREATMENT OF HOMOCYSTINURIA
HRP20251579T1 (hr) 2019-06-26 2026-01-30 Travere Therapeutics Switzerland Gmbh Pegilirana cistationin beta sintaza za enzimsku terapiju za liječenje homocistinurije
MX2022002571A (es) * 2019-09-03 2022-05-06 Travere Therapeutics Switzerland Gmbh Terapia enzimatica con cistationina beta-sintasa para el tratamiento de niveles elevados de homocisteina.
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
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

Family Cites Families (24)

* 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
US6653062B1 (en) * 2000-07-26 2003-11-25 Wisconsin Alumni Research Foundation Preservation and storage medium for biological materials
AU2003251550A1 (en) 2002-06-17 2003-12-31 The Regents Of The University Of Colorado HUMAN CYSTATHIONINE Beta-SYNTHASE VARIANTS AND METHODS OF PRODU CTION THEREOF
EP1396537A1 (en) 2002-09-04 2004-03-10 Avidis SA Treatment of elevated plasma homocysteine
KR20060065663A (ko) 2003-07-31 2006-06-14 안티캔서, 인코포레이티드 생체 내에서의 유효성 향상을 위한, ＰＥＧ-ｒＭＥＴ아제와조합된 ＰＬＰ의 용도
US20070010492A1 (en) 2005-07-11 2007-01-11 Generale Robert J Composition and method for reducing homocysteine caused by drugs containing methyl compounds
WO2011025964A2 (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
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
AU2014348683B2 (en) 2013-11-18 2020-11-05 Rubius Therapeutics, Inc. Synthetic membrane-receiver complexes
ES2865825T3 (es) 2014-04-01 2021-10-18 Rubius Therapeutics Inc Procedimientos y composiciones para inmunomodulación
US20180135012A1 (en) 2015-05-13 2018-05-17 Rubius Therapeutics, Inc. Membrane-receiver complex therapeutics
EP3373922B1 (en) 2015-11-09 2022-01-05 The Regents of The University of Colorado, A Body Corporate Compositions and methods for use in the treatment of homocystinuria
BR112019000195A2 (pt) 2016-07-07 2019-04-24 Rubius Therapeutics, Inc. composições e métodos relacionados a sistemas celulares terapêuticos que expressam rna exógeno
EP3612214A4 (en) 2017-04-17 2021-01-20 The Regents of the University of Colorado, A Body Corporate ENZYME REPLACEMENT THERAPY OPTIMIZATION FOR THE TREATMENT OF HOMOCYSTINURIA
HRP20251579T1 (hr) 2019-06-26 2026-01-30 Travere Therapeutics Switzerland Gmbh Pegilirana cistationin beta sintaza za enzimsku terapiju za liječenje homocistinurije
MX2022002571A (es) * 2019-09-03 2022-05-06 Travere Therapeutics Switzerland Gmbh Terapia enzimatica con cistationina beta-sintasa para el tratamiento de niveles elevados de homocisteina.

Also Published As

Publication number Publication date
SA521431197B1 (ar) 2024-12-03
IL289245B1 (en) 2025-05-01
FI3990005T3 (fi) 2025-11-28
AU2020302079A1 (en) 2022-02-17
CL2021003474A1 (es) 2022-09-02
IL289245A (en) 2022-02-01
EP3990005B1 (en) 2025-08-27
PH12021553235A1 (en) 2022-08-22
JP2022539077A (ja) 2022-09-07
CN114786712A (zh) 2022-07-22
MX2021015966A (es) 2022-04-06
DK3990005T3 (da) 2025-12-08
EP4663239A2 (en) 2025-12-17
US12364765B2 (en) 2025-07-22
WO2020264333A1 (en) 2020-12-30
IL289245B2 (en) 2025-09-01
US20220265835A1 (en) 2022-08-25
JP2025143275A (ja) 2025-10-01
CA3145426A1 (en) 2020-12-30
BR112021026292A2 (pt) 2022-05-31
LT3990005T (lt) 2026-01-12
PL3990005T4 (pl) 2026-03-02
PL3990005T3 (pl) 2026-03-02
PT3990005T (pt) 2025-12-17
EP3990005A1 (en) 2022-05-04
SI3990005T1 (sl) 2026-02-27
SMT202600042T1 (it) 2026-03-09
EP4663239A3 (en) 2026-03-11
MX2026001319A (es) 2026-03-02
RS67521B1 (sr) 2026-01-30
HRP20251579T1 (hr) 2026-01-30
ES3058596T3 (en) 2026-03-11

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