KR20220091464A - 상승된 호모시스테인 수준의 치료를 위한 시스타티오닌 베타-신타제 효소 치료요법 - Google Patents

상승된 호모시스테인 수준의 치료를 위한 시스타티오닌 베타-신타제 효소 치료요법 Download PDF

Info

Publication number
KR20220091464A
KR20220091464A KR1020227011039A KR20227011039A KR20220091464A KR 20220091464 A KR20220091464 A KR 20220091464A KR 1020227011039 A KR1020227011039 A KR 1020227011039A KR 20227011039 A KR20227011039 A KR 20227011039A KR 20220091464 A KR20220091464 A KR 20220091464A
Authority
KR
South Korea
Prior art keywords
levels
cbs
thcy
μmol
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
KR1020227011039A
Other languages
English (en)
Korean (ko)
Inventor
마르시아 셀로스-모라
에레즈 모세 버블릴
프랭크 글래빈
Original Assignee
트래비어 써래페틱스 스위처랜드 게엠베하
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 트래비어 써래페틱스 스위처랜드 게엠베하 filed Critical 트래비어 써래페틱스 스위처랜드 게엠베하
Publication of KR20220091464A publication Critical patent/KR20220091464A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88Lyases (4.)
    • 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
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6803General methods of protein analysis not limited to specific proteins or families of proteins
    • G01N33/6806Determination of free amino acids
    • G01N33/6812Assays for specific amino acids
    • G01N33/6815Assays for specific amino acids containing sulfur, e.g. cysteine, cystine, methionine, homocysteine
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/04Endocrine or metabolic disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/52Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Physics & Mathematics (AREA)
KR1020227011039A 2019-09-03 2020-09-03 상승된 호모시스테인 수준의 치료를 위한 시스타티오닌 베타-신타제 효소 치료요법 Pending KR20220091464A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962895230P 2019-09-03 2019-09-03
US62/895,230 2019-09-03
US202062983862P 2020-03-02 2020-03-02
US62/983,862 2020-03-02
PCT/US2020/049156 WO2021046190A1 (en) 2019-09-03 2020-09-03 Cystathionine beta-synthase enzyme therapy for treatment of elevated homocysteine levels

Publications (1)

Publication Number Publication Date
KR20220091464A true KR20220091464A (ko) 2022-06-30

Family

ID=74852233

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227011039A Pending KR20220091464A (ko) 2019-09-03 2020-09-03 상승된 호모시스테인 수준의 치료를 위한 시스타티오닌 베타-신타제 효소 치료요법

Country Status (11)

Country Link
US (1) US20220290116A1 (https=)
EP (1) EP4025243A4 (https=)
JP (2) JP2022546550A (https=)
KR (1) KR20220091464A (https=)
CN (1) CN114630673A (https=)
AU (1) AU2020343314A1 (https=)
BR (1) BR112022003539A2 (https=)
CA (1) CA3152379A1 (https=)
IL (1) IL290685A (https=)
MX (1) MX2022002571A (https=)
WO (1) WO2021046190A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FI3998067T3 (fi) 2015-11-09 2024-10-31 Univ Colorado Regents Koostumukset ja menetelmät homokystinurian hoitoon
IL269544B2 (en) 2017-04-17 2025-01-01 Univ Colorado Regents Streamlining enzyme replacement therapy for the treatment of high homocysteine
ES3058596T3 (en) * 2019-06-26 2026-03-11 Travere Therapeutics Switzerland Gmbh Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
FI3998067T3 (fi) * 2015-11-09 2024-10-31 Univ Colorado Regents Koostumukset ja menetelmät homokystinurian hoitoon
IL269544B2 (en) * 2017-04-17 2025-01-01 Univ Colorado Regents Streamlining enzyme replacement therapy for the treatment of high homocysteine
ES3058596T3 (en) * 2019-06-26 2026-03-11 Travere Therapeutics Switzerland Gmbh Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria

Also Published As

Publication number Publication date
BR112022003539A2 (pt) 2022-05-24
CA3152379A1 (en) 2021-03-11
JP2022546550A (ja) 2022-11-04
AU2020343314A1 (en) 2022-03-24
CN114630673A (zh) 2022-06-14
US20220290116A1 (en) 2022-09-15
MX2022002571A (es) 2022-05-06
IL290685A (en) 2022-04-01
JP2025183204A (ja) 2025-12-16
EP4025243A1 (en) 2022-07-13
EP4025243A4 (en) 2023-09-27
WO2021046190A1 (en) 2021-03-11

Similar Documents

Publication Publication Date Title
US12364765B2 (en) Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria
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
Pezzini et al. Homocysteine and cerebral ischemia: pathogenic and therapeutical implications
Gottlieb et al. Cardioprotection requires taking out the trash
US12233088B2 (en) Bis-choline tetrathiomolybdate for treating Wilson Disease
Rudolph et al. Coronary plaque injury triggers neutrophil activation in patients with coronary artery disease
RU2833879C1 (ru) Терапия с использованием фермента цистатионин-бета-синтазы для лечения заболеваний, вызываемых повышенными уровнями гомоцистеина
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
RU2787632C2 (ru) Бис-холина тетратиомолибдат для лечения болезни вильсона
CA3084100C (en) Bis-choline tetrathiomolybdate for treating wilson disease
Petrea et al. Homocysteine and neurological disorders
Price et al. Homocysteine: A Risk Factor for Atherothrombotic Cardiovascular Disease
Windrum Homocysteine and CVD risk: Is it relevant or redundant?

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000