CO2023007726A2 - Tratamiento para la deficiencia de enpp1 y la deficiencia de abcc6 - Google Patents

Tratamiento para la deficiencia de enpp1 y la deficiencia de abcc6

Info

Publication number
CO2023007726A2
CO2023007726A2 CONC2023/0007726A CO2023007726A CO2023007726A2 CO 2023007726 A2 CO2023007726 A2 CO 2023007726A2 CO 2023007726 A CO2023007726 A CO 2023007726A CO 2023007726 A2 CO2023007726 A2 CO 2023007726A2
Authority
CO
Colombia
Prior art keywords
deficiency
enpp1
treatment
abcc6
calcification
Prior art date
Application number
CONC2023/0007726A
Other languages
English (en)
Inventor
Pedro Huertas
Deborah Wenkert
Original Assignee
Inozyme Pharma 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 filed Critical Inozyme Pharma Inc
Publication of CO2023007726A2 publication Critical patent/CO2023007726A2/es

Links

Classifications

    • 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)
    • C12Y301/04001Phosphodiesterase I (3.1.4.1)
    • 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
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/04Drugs for skeletal disorders for non-specific disorders of the connective tissue
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system

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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

La presente descripción proporciona, entre otras cosas, dosis específicas de un agente de ENPP1 para el tratamiento in vivo de una deficiencia de ENPP1, tal como para el tratamiento de la calcificación arterial generalizada de la infancia (GACI), el raquitismo hipofosfatémico autosómico recesivo 2 (ARHR2), y otras enfermedades que resultan de la calcificación patológica, la deficiencia de ENPP1, la deficiencia de ABCC6 tales como enfermedades o trastornos que involucran la calcificación ectópica de tejido blando en un sujeto.
CONC2023/0007726A 2020-11-19 2023-06-14 Tratamiento para la deficiencia de enpp1 y la deficiencia de abcc6 CO2023007726A2 (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202063116106P 2020-11-19 2020-11-19
US202063116086P 2020-11-19 2020-11-19
US202063116093P 2020-11-19 2020-11-19
US202163219229P 2021-07-07 2021-07-07
US202163237351P 2021-08-26 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
CO2023007726A2 true CO2023007726A2 (es) 2023-07-21

Family

ID=81709698

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2023/0007726A CO2023007726A2 (es) 2020-11-19 2023-06-14 Tratamiento para la deficiencia de enpp1 y la deficiencia de abcc6

Country Status (10)

Country Link
EP (1) EP4247406A1 (es)
JP (1) JP2024500285A (es)
KR (1) KR20230123932A (es)
AU (1) AU2021383830A1 (es)
CA (1) CA3198957A1 (es)
CO (1) CO2023007726A2 (es)
IL (1) IL302954A (es)
MX (1) MX2023005836A (es)
TW (1) TW202235100A (es)
WO (1) WO2022109344A1 (es)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2899895T3 (es) * 2014-12-19 2022-03-15 Inozyme Pharma Inc Procedimientos de tratamiento de calcificación tisular
KR20210142599A (ko) * 2019-01-18 2021-11-25 이노자임 파마, 인코포레이티드 Enpp1 또는 enpp3 결핍을 수반하는 질환의 치료

Also Published As

Publication number Publication date
IL302954A (en) 2023-07-01
EP4247406A1 (en) 2023-09-27
TW202235100A (zh) 2022-09-16
MX2023005836A (es) 2023-08-17
AU2021383830A1 (en) 2023-06-22
WO2022109344A1 (en) 2022-05-27
KR20230123932A (ko) 2023-08-24
JP2024500285A (ja) 2024-01-09
CA3198957A1 (en) 2022-05-27

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