IL326528A - Treatment of enpp1 deficiency and abcc6 deficiency in children - Google Patents

Treatment of enpp1 deficiency and abcc6 deficiency in children

Info

Publication number
IL326528A
IL326528A IL326528A IL32652826A IL326528A IL 326528 A IL326528 A IL 326528A IL 326528 A IL326528 A IL 326528A IL 32652826 A IL32652826 A IL 32652826A IL 326528 A IL326528 A IL 326528A
Authority
IL
Israel
Prior art keywords
deficiency
abcc6
children
treatment
enpp1
Prior art date
Application number
IL326528A
Other languages
Hebrew (he)
Original Assignee
Inozyme Pharma Inc
Biomarin Pharm Inc
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 Inozyme Pharma Inc, Biomarin Pharm Inc filed Critical Inozyme Pharma Inc
Publication of IL326528A publication Critical patent/IL326528A/en

Links

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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
    • C12Y306/01009—Nucleotide diphosphatase (3.6.1.9), i.e. nucleotide-pyrophosphatase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL326528A 2023-08-22 2024-08-21 Treatment of enpp1 deficiency and abcc6 deficiency in children IL326528A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363578137P 2023-08-22 2023-08-22
PCT/US2024/043242 WO2025042995A1 (en) 2023-08-22 2024-08-21 Treatment of enpp1 deficiency and abcc6 deficiency in children

Publications (1)

Publication Number Publication Date
IL326528A true IL326528A (en) 2026-04-01

Family

ID=94732792

Family Applications (1)

Application Number Title Priority Date Filing Date
IL326528A IL326528A (en) 2023-08-22 2024-08-21 Treatment of enpp1 deficiency and abcc6 deficiency in children

Country Status (5)

Country Link
CN (1) CN121816188A (en)
AU (1) AU2024326788A1 (en)
CO (1) CO2026002943A2 (en)
IL (1) IL326528A (en)
WO (1) WO2025042995A1 (en)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20250078598A (en) * 2015-05-19 2025-06-02 예일 유니버시티 Compositions for treating pathological calcification conditions, and methods using same
AU2021383830A1 (en) * 2020-11-19 2023-06-22 Biomarin Pharmaceutical Inc. Treatment of enpp1 deficiency and abcc6 deficiency

Also Published As

Publication number Publication date
WO2025042995A9 (en) 2025-10-16
CN121816188A (en) 2026-04-07
WO2025042995A1 (en) 2025-02-27
AU2024326788A1 (en) 2026-03-05
CO2026002943A2 (en) 2026-04-16

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