AU2021383830A1 - Treatment of enpp1 deficiency and abcc6 deficiency - Google Patents

Treatment of enpp1 deficiency and abcc6 deficiency Download PDF

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Publication number
AU2021383830A1
AU2021383830A1 AU2021383830A AU2021383830A AU2021383830A1 AU 2021383830 A1 AU2021383830 A1 AU 2021383830A1 AU 2021383830 A AU2021383830 A AU 2021383830A AU 2021383830 A AU2021383830 A AU 2021383830A AU 2021383830 A1 AU2021383830 A1 AU 2021383830A1
Authority
AU
Australia
Prior art keywords
subject
enpp1
per kilogram
agent
calcification
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
AU2021383830A
Other languages
English (en)
Other versions
AU2021383830A9 (en
Inventor
Pedro Huertas
Deborah WENKERT
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biomarin Pharmaceutical Inc
Original Assignee
Biomarin Pharmaceutical 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 Biomarin Pharmaceutical Inc filed Critical Biomarin Pharmaceutical Inc
Publication of AU2021383830A1 publication Critical patent/AU2021383830A1/en
Publication of AU2021383830A9 publication Critical patent/AU2021383830A9/en
Assigned to BIOMARIN PHARMACEUTICAL INC. reassignment BIOMARIN PHARMACEUTICAL INC. Request for Assignment Assignors: INOZYME PHARMA, INC.
Pending legal-status Critical Current

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Classifications

    • 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)
    • 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
    • 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)
AU2021383830A 2020-11-19 2021-11-19 Treatment of enpp1 deficiency and abcc6 deficiency Pending AU2021383830A1 (en)

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 (2)

Publication Number Publication Date
AU2021383830A1 true AU2021383830A1 (en) 2023-06-22
AU2021383830A9 AU2021383830A9 (en) 2024-06-27

Family

ID=81709698

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021383830A Pending AU2021383830A1 (en) 2020-11-19 2021-11-19 Treatment of enpp1 deficiency and abcc6 deficiency

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
KR20230123932A (ko) 2023-08-24
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
CA3198957A1 (en) 2022-05-27
JP2024500285A (ja) 2024-01-09
IL302954A (en) 2023-07-01
MX2023005836A (es) 2023-08-17

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Legal Events

Date Code Title Description
SREP Specification republished
PC1 Assignment before grant (sect. 113)

Owner name: BIOMARIN PHARMACEUTICAL INC.

Free format text: FORMER APPLICANT(S): INOZYME PHARMA, INC.