KR102769578B1 - 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료 - Google Patents

산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료 Download PDF

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KR102769578B1
KR102769578B1 KR1020207008213A KR20207008213A KR102769578B1 KR 102769578 B1 KR102769578 B1 KR 102769578B1 KR 1020207008213 A KR1020207008213 A KR 1020207008213A KR 20207008213 A KR20207008213 A KR 20207008213A KR 102769578 B1 KR102769578 B1 KR 102769578B1
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patient
pharmaceutical composition
leu
acid sphingomyelinase
bone
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KR20200044064A (ko
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아나 크리스티나 샤이트-푸가
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사노피
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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
    • A61K38/46—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
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • 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
    • 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)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
KR1020207008213A 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료 Active KR102769578B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020257004704A KR20250025051A (ko) 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762549732P 2017-08-24 2017-08-24
US62/549,732 2017-08-24
EP17306720 2017-12-07
EP17306720.8 2017-12-07
PCT/IB2018/056346 WO2019038685A2 (en) 2017-08-24 2018-08-22 TREATMENT OF ABNORMAL BONE CONDITIONS IN PATIENTS WITH ACIDIC SPHINGOMYELASE DISEASE

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020257004704A Division KR20250025051A (ko) 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

Publications (2)

Publication Number Publication Date
KR20200044064A KR20200044064A (ko) 2020-04-28
KR102769578B1 true KR102769578B1 (ko) 2025-02-20

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KR1020207008213A Active KR102769578B1 (ko) 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료
KR1020257004704A Pending KR20250025051A (ko) 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

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KR1020257004704A Pending KR20250025051A (ko) 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

Country Status (16)

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US (2) US11898175B2 (enExample)
EP (1) EP3672622B1 (enExample)
JP (3) JP7216075B2 (enExample)
KR (2) KR102769578B1 (enExample)
CN (2) CN118453843A (enExample)
AU (2) AU2018319565B2 (enExample)
BR (1) BR112020003541A2 (enExample)
CA (1) CA3073648A1 (enExample)
CO (1) CO2020001801A2 (enExample)
ES (1) ES2970423T3 (enExample)
IL (1) IL272757B2 (enExample)
MX (2) MX2020002106A (enExample)
PL (1) PL3672622T3 (enExample)
SG (1) SG11202001544VA (enExample)
TW (1) TWI791040B (enExample)
WO (1) WO2019038685A2 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2773133A1 (en) * 2009-08-28 2011-03-03 Edward H. Schuchman Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
PL3672622T3 (pl) * 2017-08-24 2024-04-02 Genzyme Corporation Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy
PY1939780A (es) * 2018-05-25 2019-12-04 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida
JP7568648B2 (ja) * 2019-05-31 2024-10-16 ジェンザイム・コーポレーション 二次元ｌｃ－ｍｓ／ｍｓシステム
CN112773779B (zh) * 2021-02-04 2022-08-16 上海交通大学医学院附属新华医院 氨溴索、千金藤和汉防已在治疗酸性鞘磷脂酶缺乏症中的应用
TW202403043A (zh) * 2022-03-18 2024-01-16 美商健臻公司 重組人類酸性鞘磷脂酶醫藥組合物及方法

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
CN1302897A (zh) 1999-10-28 2001-07-11 上海博容基因开发有限公司 一种新的多肽——人酸性鞘磷酯酶相似的磷酸二酯酶21和编码这种多肽的多核苷酸
US8158582B2 (en) * 2005-06-02 2012-04-17 Tercica, Inc. Methods for treatment of insulin-like growth factor-1 deficiency
CA2773133A1 (en) * 2009-08-28 2011-03-03 Edward H. Schuchman Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
WO2013130963A1 (en) 2012-03-02 2013-09-06 Shire Human Genetic Therapies, Inc. Compositions and methods for treating type iii gaucher disease
AR093908A1 (es) 2012-12-12 2015-06-24 Teva Pharma Fusion de la hormona de crecimiento humana y albumina, formulacion y usos de la misma
MX370570B (es) * 2013-06-07 2019-12-17 Genzyme Corp Marcador para trastornos de la esfingomielinasa acida y sus usos.
PL3672622T3 (pl) * 2017-08-24 2024-04-02 Genzyme Corporation Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy

Also Published As

Publication number Publication date
CN118453843A (zh) 2024-08-09
JP2023052459A (ja) 2023-04-11
AU2018319565B2 (en) 2025-02-20
BR112020003541A2 (pt) 2020-09-01
WO2019038685A2 (en) 2019-02-28
MX2020002106A (es) 2020-07-14
AU2018319565A1 (en) 2020-04-09
IL272757B2 (en) 2024-07-01
NZ762860A (en) 2025-09-26
TWI791040B (zh) 2023-02-01
RU2020111649A (ru) 2021-09-24
JP2024050732A (ja) 2024-04-10
IL272757A (en) 2020-04-30
ES2970423T3 (es) 2024-05-28
IL272757B1 (en) 2024-03-01
US20240218338A1 (en) 2024-07-04
PL3672622T3 (pl) 2024-04-02
CA3073648A1 (en) 2019-02-28
JP7431356B2 (ja) 2024-02-14
EP3672622A2 (en) 2020-07-01
WO2019038685A3 (en) 2019-05-02
MX2023010908A (es) 2023-09-27
SG11202001544VA (en) 2020-03-30
JP2020531527A (ja) 2020-11-05
TW201919689A (zh) 2019-06-01
US20210139868A1 (en) 2021-05-13
JP7216075B2 (ja) 2023-01-31
EP3672622B1 (en) 2023-11-08
CN111344003B (zh) 2024-05-07
KR20250025051A (ko) 2025-02-20
CN111344003A (zh) 2020-06-26
AU2025203384A1 (en) 2025-05-29
KR20200044064A (ko) 2020-04-28
US11898175B2 (en) 2024-02-13
CO2020001801A2 (es) 2020-04-01

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