MX2021015966A - Producto farmacológico para terapia enzimática para el tratamiento de la homocistinuria. - Google Patents

Producto farmacológico para terapia enzimática para el tratamiento de la homocistinuria.

Info

Publication number
MX2021015966A
MX2021015966A MX2021015966A MX2021015966A MX2021015966A MX 2021015966 A MX2021015966 A MX 2021015966A MX 2021015966 A MX2021015966 A MX 2021015966A MX 2021015966 A MX2021015966 A MX 2021015966A MX 2021015966 A MX2021015966 A MX 2021015966A
Authority
MX
Mexico
Prior art keywords
homocystinuria
treatment
pegylated
cystathionine beta
enzyme therapy
Prior art date
Application number
MX2021015966A
Other languages
English (en)
Spanish (es)
Inventor
Tomas Majtan
Erez Bublil
Frank Glavin
Marcia Sellos-Moura
Kraus, (Fallecido) Jan P
Randy Wanner
Orhan Causevic
Original Assignee
Travere Therapeutics Switzerland Gmbh
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 filed Critical Travere Therapeutics Switzerland Gmbh
Publication of MX2021015966A publication Critical patent/MX2021015966A/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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
    • A61K47/59Medicinal 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
    • A61K47/60Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51Lyases (4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02Inorganic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/08Drugs for disorders of the alimentary tract or the digestive system for nausea, cinetosis or vertigo; Antiemetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y402/00Carbon-oxygen lyases (4.2)
    • C12Y402/01Hydro-lyases (4.2.1)
    • C12Y402/01022Cystathionine 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)
MX2021015966A 2019-06-26 2020-06-26 Producto farmacológico para terapia enzimática para el tratamiento de la homocistinuria. MX2021015966A (es)

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
MX2021015966A true MX2021015966A (es) 2022-04-06

Family

ID=71670437

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2021015966A MX2021015966A (es) 2019-06-26 2020-06-26 Producto farmacológico para terapia enzimática para el tratamiento de la homocistinuria.
MX2026001319A MX2026001319A (es) 2019-06-26 2021-12-16 Producto farmacologico para terapia enzimatica para el tratamiento de la homocistinuria

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2026001319A MX2026001319A (es) 2019-06-26 2021-12-16 Producto farmacologico para terapia enzimatica para el tratamiento de la homocistinuria

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
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
KR20220044726A (ko) 2022-04-11
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

Similar Documents

Publication Publication Date Title
MX2026001319A (es) Producto farmacologico para terapia enzimatica para el tratamiento de la homocistinuria
PH12020550258A1 (en) Trispecific proteins and methods of use
MX2021007392A (es) Regimen de dosificacion y combinacion farmaceutica que comprende derivados de 3-(1-oxoisoindolin-2-il)piperidina-2,6-diona.
MX2023001466A (es) Enlazadores de base peptidica.
NZ605871A (en) Treatment of sanfilippo syndrome type b
NZ702800A (en) Methods and compositions for cns delivery of heparan n-sulfatase
NZ587105A (en) Tissue protective peptides and peptide analogs for preventing and treating diseases and disorders associated with tissue damage
MX363136B (es) Composicion farmaceutica para el tratamiento y/o prevencion del cancer.
MX360211B (es) Composicion farmaceutica para el tratamiento y/o prevencion del cancer.
CL2020002704A1 (es) Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial
NZ605874A (en) Methods and compositions for cns delivery of heparan n-sulfatase
HK1249433A1 (zh) 含有mk2抑制剂肽的组合物用於治疗非小细胞肺癌的用途
WO2022256500A3 (en) Dll3 targeting trispecific proteins and methods of use
GEP20257840B (en) Tyk2 pseudokinase ligands
NZ758331A (en) B cells for in vivo delivery of therapeutic agents and dosages thereof
EA201990885A1 (ru) Композиции, содержащие короткие пептиды, полученные из pedf, и их применение
CA3059592A1 (en) Optimization of enzyme replacement therapy for treatment of homocystinuria
MX2022004944A (es) Composiciones y metodos para minimizar la perdida de proteinas a concentraciones de proteinas bajas.
MX2022005506A (es) Formulaciones y dosis de uricasa pegilada.
MX2022007546A (es) Interferon-alfa-2 modificado que tiene inmunogenicidad reducida.
ZA202207211B (en) Peptide docking vehicle for targeted nucleic acid delivery
CN104968673A (zh) 融合多肽及使用方法
EP4487914A2 (en) Compositions and methods for treatment of homocystinuria
NZ603442A (en) Treatment of proliferative diseases
EA202192328A1 (ru) Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления