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SA521431197B1 - منتج دوائي للعلاج بالإنزيم لعلاج البيلة الهوموسيستينية (اضطراب في الهوموسيستين) - Google Patents

منتج دوائي للعلاج بالإنزيم لعلاج البيلة الهوموسيستينية (اضطراب في الهوموسيستين)

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Publication number
SA521431197B1
SA521431197B1 SA521431197A SA521431197A SA521431197B1 SA 521431197 B1 SA521431197 B1 SA 521431197B1 SA 521431197 A SA521431197 A SA 521431197A SA 521431197 A SA521431197 A SA 521431197A SA 521431197 B1 SA521431197 B1 SA 521431197B1
Authority
SA
Saudi Arabia
Prior art keywords
homocystinuria
treatment
homocysteine
disorder
enzyme therapy
Prior art date
Application number
SA521431197A
Other languages
English (en)
Inventor
جان بي. كراوس،
فرانك جلافين،
، مارسيا سيلوس-مورا
اورهان كاوسفيك،
راندي وانر،
إيرز ببليل،
توماس ماجتان،
Original Assignee
ترافير ثيرابيوتكس سويتزرلاند جي ام بي اتش
ذي ريجينتس اوف يونيفيرسيتي اوف كولورادو
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 ترافير ثيرابيوتكس سويتزرلاند جي ام بي اتش, ذي ريجينتس اوف يونيفيرسيتي اوف كولورادو filed Critical ترافير ثيرابيوتكس سويتزرلاند جي ام بي اتش
Publication of SA521431197B1 publication Critical patent/SA521431197B1/ar

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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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Inorganic Chemistry (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Otolaryngology (AREA)
  • Hematology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Neurosurgery (AREA)
  • Obesity (AREA)
  • Biomedical Technology (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

منتج دوائي للعلاج بالإنزيم لعلاج البيلة الهوموسيستينية (اضطراب في الهوموسيستين) DRUG PRODUCT FOR ENZYME THERAPY FOR TREATMENT OF HOMOCYSTINURIA الملخـــص يتعلق الاختراع الحالي بصيغ لمنتج دوائي drug product مشتمل على بروتين PEGylated CBS (مُعالج بالبولي إيثيلين جليكول) له تتابع الأحماض الأمينية amino acid sequence من بيان تتابع رقم: 1. يتم توفير الجرعات Dosages وأنظمة الجرعات dosing regimen لعلاج البيلة الهوموسيستينية homocystinuria في خاضع بحاجة إلى ذلك. بالإضافة إلى ذلك، يتم أيضًا توفير الجرعات وأنظمة الجرعات لتقليل مستوى الهوموسيستين homocysteine (Hcy) أو زيادة مستويات السيستين cysteine (Cys) و/أو السيستاثيونين cystathionine (Cth) في خاضع بحاجة إلى ذلك. شكل. 3
SA521431197A 2019-06-26 2021-12-23 منتج دوائي للعلاج بالإنزيم لعلاج البيلة الهوموسيستينية (اضطراب في الهوموسيستين) SA521431197B1 (ar)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962866810P 2019-06-26 2019-06-26
US202062983860P 2020-03-02 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
SA521431197B1 true SA521431197B1 (ar) 2024-12-03

Family

ID=71670437

Family Applications (1)

Application Number Title Priority Date Filing Date
SA521431197A SA521431197B1 (ar) 2019-06-26 2021-12-23 منتج دوائي للعلاج بالإنزيم لعلاج البيلة الهوموسيستينية (اضطراب في الهوموسيستين)

Country Status (13)

Country Link
EP (1) EP3990005A1 (ar)
JP (1) JP2022539077A (ar)
KR (1) KR20220044726A (ar)
CN (1) CN114786712A (ar)
AU (1) AU2020302079A1 (ar)
BR (1) BR112021026292A2 (ar)
CA (1) CA3145426A1 (ar)
CL (1) CL2021003474A1 (ar)
IL (1) IL289245B1 (ar)
MX (1) MX2021015966A (ar)
PH (1) PH12021553235A1 (ar)
SA (1) SA521431197B1 (ar)
WO (1) WO2020264333A1 (ar)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11324811B2 (en) 2017-04-17 2022-05-10 The Regents Of The University Of Colorado, A Body Corporate Optimization of enzyme replacement therapy for treatment of homocystinuria
BR112022003539A2 (pt) * 2019-09-03 2022-05-24 Travere Therapeutics Switzerland Gmbh Terapia enzimática com cistationina beta-sintase para tratamento de níveis elevados de homocisteína
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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112012004546A8 (pt) * 2009-08-29 2017-12-05 Abbott Lab Terapêutica por proteínas ligantes dll4-ligantes
EP2510100B1 (en) * 2009-12-09 2017-10-11 CureVac AG Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
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
RU2020136119A (ru) 2014-04-01 2021-05-17 Рубиус Терапьютикс, Инк. Способы и композиции для иммуномодуляции
US20180135012A1 (en) 2015-05-13 2018-05-17 Rubius Therapeutics, Inc. Membrane-receiver complex therapeutics
MX2018005312A (es) * 2015-11-09 2018-08-15 Univ Colorado Regents Composiciones y metodos para el tratamiento de la homocistinuria.
CA3029906A1 (en) 2016-07-07 2018-01-11 Rubius Therapeutics, Inc. Compositions and methods related to therapeutic cell systems expressing exogenous rna
BR112022003539A2 (pt) * 2019-09-03 2022-05-24 Travere Therapeutics Switzerland Gmbh Terapia enzimática com cistationina beta-sintase para tratamento de níveis elevados de homocisteína

Also Published As

Publication number Publication date
KR20220044726A (ko) 2022-04-11
EP3990005A1 (en) 2022-05-04
US20220265835A1 (en) 2022-08-25
CN114786712A (zh) 2022-07-22
MX2021015966A (es) 2022-04-06
JP2022539077A (ja) 2022-09-07
PH12021553235A1 (en) 2022-08-22
IL289245A (en) 2022-02-01
WO2020264333A1 (en) 2020-12-30
CL2021003474A1 (es) 2022-09-02
BR112021026292A2 (pt) 2022-05-31
CA3145426A1 (en) 2020-12-30
AU2020302079A1 (en) 2022-02-17
IL289245B1 (en) 2025-05-01

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