KR20230123932A - Enpp1 결핍증 및 abcc6 결핍증의 치료 - Google Patents

Enpp1 결핍증 및 abcc6 결핍증의 치료 Download PDF

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Publication number
KR20230123932A
KR20230123932A KR1020237017319A KR20237017319A KR20230123932A KR 20230123932 A KR20230123932 A KR 20230123932A KR 1020237017319 A KR1020237017319 A KR 1020237017319A KR 20237017319 A KR20237017319 A KR 20237017319A KR 20230123932 A KR20230123932 A KR 20230123932A
Authority
KR
South Korea
Prior art keywords
subject
enpp1
per kilogram
leu
ser
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
KR1020237017319A
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English (en)
Korean (ko)
Inventor
페드로 후에르타스
데보라 벤커트
Original Assignee
이노자임 파마, 인코포레이티드
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
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Application filed by 이노자임 파마, 인코포레이티드 filed Critical 이노자임 파마, 인코포레이티드
Publication of KR20230123932A publication Critical patent/KR20230123932A/ko
Pending legal-status Critical Current

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Classifications

    • 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
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • A61P19/04—Drugs for skeletal disorders for non-specific disorders of the connective tissue
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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)
    • C12Y301/04001—Phosphodiesterase I (3.1.4.1)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Biotechnology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Molecular Biology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
KR1020237017319A 2020-11-19 2021-11-19 Enpp1 결핍증 및 abcc6 결핍증의 치료 Pending KR20230123932A (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063116106P 2020-11-19 2020-11-19
US202063116093P 2020-11-19 2020-11-19
US202063116086P 2020-11-19 2020-11-19
US63/116,086 2020-11-19
US63/116,106 2020-11-19
US63/116,093 2020-11-19
US202163219229P 2021-07-07 2021-07-07
US63/219,229 2021-07-07
US202163237351P 2021-08-26 2021-08-26
US63/237,351 2021-08-26
PCT/US2021/060207 WO2022109344A1 (en) 2020-11-19 2021-11-19 Treatment of enpp1 deficiency and abcc6 deficiency

Publications (1)

Publication Number Publication Date
KR20230123932A true KR20230123932A (ko) 2023-08-24

Family

ID=81709698

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237017319A Pending KR20230123932A (ko) 2020-11-19 2021-11-19 Enpp1 결핍증 및 abcc6 결핍증의 치료

Country Status (11)

Country Link
US (1) US20240181021A1 (https=)
EP (1) EP4247406A4 (https=)
JP (1) JP2024500285A (https=)
KR (1) KR20230123932A (https=)
AU (1) AU2021383830A1 (https=)
CA (1) CA3198957A1 (https=)
CO (1) CO2023007726A2 (https=)
IL (1) IL302954A (https=)
MX (1) MX2023005836A (https=)
TW (1) TW202235100A (https=)
WO (1) WO2022109344A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023196820A2 (en) * 2022-04-04 2023-10-12 Inozyme Pharma, Inc. Treatment of enpp1 deficiency and abcc6 deficiency
WO2025042995A1 (en) * 2023-08-22 2025-02-27 Inozyme Pharma, Inc. Treatment of enpp1 deficiency and abcc6 deficiency in children

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3967755B1 (en) * 2014-12-19 2024-03-27 Inozyme Pharma, Inc. Soluble npp1 for use in a method for treating pseudoxanthoma elasticum
EP4414028A3 (en) * 2015-05-19 2024-11-27 Yale University Compositions for treating pathological calcification conditions, and methods using same
CA3126839A1 (en) * 2019-01-18 2020-07-23 Inozyme Pharma, Inc. Treatment of diseases involving deficiency of enpp1 or enpp3

Also Published As

Publication number Publication date
EP4247406A4 (en) 2024-10-30
US20240181021A1 (en) 2024-06-06
TW202235100A (zh) 2022-09-16
CO2023007726A2 (es) 2023-07-21
EP4247406A1 (en) 2023-09-27
WO2022109344A1 (en) 2022-05-27
AU2021383830A1 (en) 2023-06-22
CA3198957A1 (en) 2022-05-27
JP2024500285A (ja) 2024-01-09
IL302954A (en) 2023-07-01
MX2023005836A (es) 2023-08-17

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