CN119183382A - 治疗、改善和/或防止弥漫性特发性骨肥厚症(dish)的方法和组合物 - Google Patents

治疗、改善和/或防止弥漫性特发性骨肥厚症(dish)的方法和组合物 Download PDF

Info

Publication number
CN119183382A
CN119183382A CN202280096064.9A CN202280096064A CN119183382A CN 119183382 A CN119183382 A CN 119183382A CN 202280096064 A CN202280096064 A CN 202280096064A CN 119183382 A CN119183382 A CN 119183382A
Authority
CN
China
Prior art keywords
enpp1
patient
subject
seq
per kilogram
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
CN202280096064.9A
Other languages
English (en)
Chinese (zh)
Inventor
D·布拉多克
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.)
Yale University
Original Assignee
Yale University
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 Yale University filed Critical Yale University
Publication of CN119183382A publication Critical patent/CN119183382A/zh
Pending legal-status Critical Current

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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/643—Albumins, e.g. HSA, BSA, ovalbumin or a Keyhole Limpet Hemocyanin [KHL]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/66—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid the modifying agent being a pre-targeting system involving a peptide or protein for targeting specific cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • A61K47/6815—Enzymes
    • 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
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • 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
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/036—Fusion polypeptide containing a localisation/targetting motif targeting to the medium outside of the cell, e.g. type III secretion
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • 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
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Rheumatology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Plant Substances (AREA)
CN202280096064.9A 2022-03-30 2022-09-30 治疗、改善和/或防止弥漫性特发性骨肥厚症(dish)的方法和组合物 Pending CN119183382A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263325554P 2022-03-30 2022-03-30
US63/325,554 2022-03-30
PCT/US2022/077407 WO2023191898A1 (en) 2022-03-30 2022-09-30 Method and compositions for treatment, amelioration, and/or prevention of diffuse idiopathic skeletal hyperostosis (dish)

Publications (1)

Publication Number Publication Date
CN119183382A true CN119183382A (zh) 2024-12-24

Family

ID=88202983

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280096064.9A Pending CN119183382A (zh) 2022-03-30 2022-09-30 治疗、改善和/或防止弥漫性特发性骨肥厚症(dish)的方法和组合物

Country Status (12)

Country Link
US (1) US20260041742A1 (enExample)
EP (1) EP4499130A4 (enExample)
JP (1) JP2025510963A (enExample)
KR (1) KR20250047645A (enExample)
CN (1) CN119183382A (enExample)
AU (1) AU2022450370A1 (enExample)
CA (1) CA3246877A1 (enExample)
CO (1) CO2024014833A2 (enExample)
IL (1) IL315907A (enExample)
MX (1) MX2024011876A (enExample)
TW (1) TW202342103A (enExample)
WO (1) WO2023191898A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026102337A1 (en) 2024-11-08 2026-05-15 Biomarin Pharmaceutical Inc. Enpp1 for use in the treatment of calciphylaxis
CN120163825B (zh) * 2025-05-20 2025-08-08 青岛大学 一种颈椎后纵韧带骨化症自动定位及分型方法

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1044468A1 (zh) * 1998-12-03 2002-10-25 海斯塔泰克有限责任公司 治療哮喘和炎症的小肽和方法
EP1736553A1 (en) * 2005-06-17 2006-12-27 Centre National De La Recherche Scientifique ENPP1 (PC-1) gene haplotype associated with the risk of obesity and type 2 diabetes and their applications
JP2021523139A (ja) * 2018-05-08 2021-09-02 イエール ユニバーシティ 腎石症の進行を遅らせるための組成物および方法
EP3844280A4 (en) * 2018-08-31 2022-09-14 Yale University ENPP1 POLYPEPTIDES AND METHODS OF USE
US20220195402A1 (en) * 2019-04-05 2022-06-23 Yale University Enpp1 polypeptides and methods of using same
EP4162036A1 (en) * 2020-06-09 2023-04-12 Inozyme Pharma, Inc. Soluble enpp1 or enpp3 proteins and uses thereof

Also Published As

Publication number Publication date
CA3246877A1 (en) 2023-10-05
CO2024014833A2 (es) 2025-01-23
EP4499130A1 (en) 2025-02-05
JP2025510963A (ja) 2025-04-15
EP4499130A4 (en) 2026-03-04
AU2022450370A1 (en) 2024-10-24
MX2024011876A (es) 2025-01-09
WO2023191898A1 (en) 2023-10-05
KR20250047645A (ko) 2025-04-04
IL315907A (en) 2024-11-01
TW202342103A (zh) 2023-11-01
US20260041742A1 (en) 2026-02-12

Similar Documents

Publication Publication Date Title
RU2754558C2 (ru) Лечение мышечной слабости с помощью щелочных фосфатаз
JP2007533669A (ja) 骨送達複合体ならびにタンパク質に骨を標的化させるためのその使用方法
US20240181021A1 (en) Treatment of ENPP1 Deficiency and ABCC6 Deficiency
US20260041742A1 (en) Method and compositions for treatment, amelioration, and/or prevention of diffuse idiopathic skeletal hyperostosis (dish)
US20250170223A1 (en) Treatment of enpp1 deficiency and abcc6 deficiency
HK40120234A (en) Method and compositions for treatment, amelioration, and/or prevention of diffuse idiopathic skeletal hyperostosis (dish)
BR112024019927B1 (pt) Uso de um composto para melhorar e/ou prevenir a hiperostose esquelética idiopática difusa (dish
KR20260058843A (ko) 소아 enpp1 결핍증 및 abcc6 결핍증의 치료
CN116710122A (zh) Enpp1缺乏症和abcc6缺乏症的治疗
HK40093022A (en) Treatment of enpp1 deficiency and abcc6 deficiency
WO2025255052A1 (en) Treatment of enpp1 deficiency and abcc6 deficiency in infants
JP2026516225A (ja) アルカリホスファターゼによる筋力低下の処置
US20250073319A1 (en) Lyophilized enpp1 polypeptide formulations and uses thereof
WO2026102337A1 (en) Enpp1 for use in the treatment of calciphylaxis
HK40112655A (en) Lyophilized enpp1 polypeptide formulations and uses thereof
WO2003012120A2 (en) Lp354 mammalian secreted protein

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40120234

Country of ref document: HK