KR102094253B1 - 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 - Google Patents

산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 Download PDF

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KR102094253B1
KR102094253B1 KR1020127005293A KR20127005293A KR102094253B1 KR 102094253 B1 KR102094253 B1 KR 102094253B1 KR 1020127005293 A KR1020127005293 A KR 1020127005293A KR 20127005293 A KR20127005293 A KR 20127005293A KR 102094253 B1 KR102094253 B1 KR 102094253B1
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에드워드 에이치 슈크맨
로버트 제이 데스닉
제랄드 에프 콕스
로라 피 앤드류스
제임스 엠 머레이
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이칸 스쿨 오브 메디슨 엣 마운트 시나이
젠자임 코포레이션
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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
    • 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
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)

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  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
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  • Genetics & Genomics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pulmonology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Dermatology (AREA)
KR1020127005293A 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 Active KR102094253B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US23811309P 2009-08-28 2009-08-28
US61/238,113 2009-08-28
PCT/US2010/047057 WO2011025996A2 (en) 2009-08-28 2010-08-28 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

Related Child Applications (1)

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KR1020207008342A Division KR20200034828A (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법

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Publication Number Publication Date
KR20120081077A KR20120081077A (ko) 2012-07-18
KR102094253B1 true KR102094253B1 (ko) 2020-03-31

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KR1020127005293A Active KR102094253B1 (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020257039475A Pending KR20250171458A (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020227023712A Active KR102608551B1 (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020207008342A Ceased KR20200034828A (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020237040891A Active KR102893390B1 (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020217026374A Active KR102608546B1 (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법

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KR1020257039475A Pending KR20250171458A (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020227023712A Active KR102608551B1 (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020207008342A Ceased KR20200034828A (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020237040891A Active KR102893390B1 (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
KR1020217026374A Active KR102608546B1 (ko) 2009-08-28 2010-08-28 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법

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US (9) US8349319B2 (enExample)
EP (4) EP2470199B1 (enExample)
JP (8) JP5955219B2 (enExample)
KR (6) KR102094253B1 (enExample)
CN (3) CN105999238A (enExample)
AU (6) AU2010286550B2 (enExample)
BR (1) BR112012004377A2 (enExample)
CA (1) CA2773133A1 (enExample)
CL (1) CL2012000495A1 (enExample)
CY (1) CY1122128T1 (enExample)
DK (3) DK3482767T3 (enExample)
ES (3) ES3052684T3 (enExample)
FI (2) FI3998078T3 (enExample)
FR (1) FR22C1062I2 (enExample)
HR (3) HRP20251310T1 (enExample)
HU (3) HUE057440T2 (enExample)
IL (4) IL314807A (enExample)
LT (4) LT3998078T (enExample)
LU (1) LUC00288I2 (enExample)
MX (2) MX356873B (enExample)
NL (1) NL301211I2 (enExample)
NO (1) NO2022054I1 (enExample)
PL (3) PL2470199T3 (enExample)
PT (3) PT3482767T (enExample)
RU (2) RU2731616C2 (enExample)
SI (3) SI2470199T1 (enExample)
SM (1) SMT202500432T1 (enExample)
TR (1) TR201911293T4 (enExample)
WO (1) WO2011025996A2 (enExample)
ZA (2) ZA201201435B (enExample)

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