CA3145426A1 - Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria - Google Patents

Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria Download PDF

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Publication number
CA3145426A1
CA3145426A1 CA3145426A CA3145426A CA3145426A1 CA 3145426 A1 CA3145426 A1 CA 3145426A1 CA 3145426 A CA3145426 A CA 3145426A CA 3145426 A CA3145426 A CA 3145426A CA 3145426 A1 CA3145426 A1 CA 3145426A1
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CA
Canada
Prior art keywords
hereby incorporated
entirety
see
hcy
patients
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
CA3145426A
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English (en)
French (fr)
Inventor
Jan P. Kraus (Deceased)
Randy WANNER
Erez Bublil
Frank GLAVIN
Marcia Sellos-Moura
Tomas Majtan
Orhan CAUSEVIC
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 CA3145426A1 publication Critical patent/CA3145426A1/en
Pending legal-status Critical Current

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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)
  • 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)
CA3145426A 2019-06-26 2020-06-26 Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria Pending CA3145426A1 (en)

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
CA3145426A1 true CA3145426A1 (en) 2020-12-30

Family

ID=71670437

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3145426A Pending CA3145426A1 (en) 2019-06-26 2020-06-26 Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria

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

Family Cites Families (24)

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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
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
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
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

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
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
JP2022539077A (ja) 2022-09-07

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