KR20250025051A - 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료 - Google Patents

산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료 Download PDF

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Publication number
KR20250025051A
KR20250025051A KR1020257004704A KR20257004704A KR20250025051A KR 20250025051 A KR20250025051 A KR 20250025051A KR 1020257004704 A KR1020257004704 A KR 1020257004704A KR 20257004704 A KR20257004704 A KR 20257004704A KR 20250025051 A KR20250025051 A KR 20250025051A
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South Korea
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leu
pro
ala
gly
patient
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Pending
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KR1020257004704A
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English (en)
Korean (ko)
Inventor
아나 크리스티나 샤이트-푸가
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사노피
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Publication of KR20250025051A publication Critical patent/KR20250025051A/ko
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    • 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
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • 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
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
KR1020257004704A 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료 Pending KR20250025051A (ko)

Applications Claiming Priority (6)

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
KR1020207008213A KR102769578B1 (ko) 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료
PCT/IB2018/056346 WO2019038685A2 (en) 2017-08-24 2018-08-22 TREATMENT OF ABNORMAL BONE CONDITIONS IN PATIENTS WITH ACIDIC SPHINGOMYELASE DISEASE

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020207008213A Division KR102769578B1 (ko) 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

Publications (1)

Publication Number Publication Date
KR20250025051A true KR20250025051A (ko) 2025-02-20

Family

ID=63556374

Family Applications (2)

Application Number Title Priority Date Filing Date
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

Family Applications Before (1)

Application Number Title Priority Date Filing Date
KR1020207008213A Active KR102769578B1 (ko) 2017-08-24 2018-08-22 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

Country Status (17)

Country Link
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)
NZ (1) NZ762860A (enExample)
PL (1) PL3672622T3 (enExample)
SG (1) SG11202001544VA (enExample)
TW (1) TWI791040B (enExample)
WO (1) WO2019038685A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112012004377A2 (pt) * 2009-08-28 2017-12-12 Genzyme Corp terapia de reposição enzimática com progressão de dose para tratamento de deficiência de esfingomielinase ácida.
UY38238A (es) * 2018-05-25 2019-12-31 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida
HUE071194T2 (hu) * 2019-05-31 2025-08-28 Genzyme Corp Kétdimenziós LC-MS/MS rendszerek
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

Family Cites Families (7)

* 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
ES2532089T3 (es) * 2005-06-02 2015-03-24 Ipsen Biopharmaceuticals, Inc. GH e IGF-1 para el tratamiento de trastornos del crecimiento
BR112012004377A2 (pt) * 2009-08-28 2017-12-12 Genzyme Corp terapia de reposição enzimática com progressão de dose para tratamento de deficiência de esfingomielinase ácida.
KR102096752B1 (ko) 2012-03-02 2020-04-02 샤이어 휴먼 지네틱 테라피즈 인크. Iii형 고셔병을 치료하기 위한 조성물 및 방법
EA201591108A1 (ru) 2012-12-12 2015-11-30 Тева Фармасьютикал Индастриз Лтд. Слитый гормон роста человека и альбумин, составы и их применения
KR102228367B1 (ko) 2013-06-07 2021-03-16 젠자임 코포레이션 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도

Also Published As

Publication number Publication date
AU2018319565A1 (en) 2020-04-09
EP3672622A2 (en) 2020-07-01
KR20200044064A (ko) 2020-04-28
KR102769578B1 (ko) 2025-02-20
JP2024050732A (ja) 2024-04-10
US20210139868A1 (en) 2021-05-13
SG11202001544VA (en) 2020-03-30
US20240218338A1 (en) 2024-07-04
JP7216075B2 (ja) 2023-01-31
MX2020002106A (es) 2020-07-14
TW201919689A (zh) 2019-06-01
WO2019038685A2 (en) 2019-02-28
WO2019038685A3 (en) 2019-05-02
IL272757A (en) 2020-04-30
CN118453843A (zh) 2024-08-09
US11898175B2 (en) 2024-02-13
EP3672622B1 (en) 2023-11-08
PL3672622T3 (pl) 2024-04-02
MX2023010908A (es) 2023-09-27
JP2023052459A (ja) 2023-04-11
TWI791040B (zh) 2023-02-01
JP2020531527A (ja) 2020-11-05
BR112020003541A2 (pt) 2020-09-01
ES2970423T3 (es) 2024-05-28
IL272757B1 (en) 2024-03-01
IL272757B2 (en) 2024-07-01
AU2025203384A1 (en) 2025-05-29
AU2018319565B2 (en) 2025-02-20
NZ762860A (en) 2025-09-26
CN111344003B (zh) 2024-05-07
CO2020001801A2 (es) 2020-04-01
RU2020111649A (ru) 2021-09-24
CN111344003A (zh) 2020-06-26
JP7431356B2 (ja) 2024-02-14
CA3073648A1 (en) 2019-02-28

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Comment text: Divisional Application for International Patent

Patent event code: PA01041R01D

Patent event date: 20250213

Application number text: 1020207008213

Filing date: 20200320

PA0201 Request for examination
PG1501 Laying open of application