CA3198957A1 - Treatment of enpp1 deficiency and abcc6 deficiency - Google Patents

Treatment of enpp1 deficiency and abcc6 deficiency

Info

Publication number
CA3198957A1
CA3198957A1 CA3198957A CA3198957A CA3198957A1 CA 3198957 A1 CA3198957 A1 CA 3198957A1 CA 3198957 A CA3198957 A CA 3198957A CA 3198957 A CA3198957 A CA 3198957A CA 3198957 A1 CA3198957 A1 CA 3198957A1
Authority
CA
Canada
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
CA3198957A
Other languages
English (en)
French (fr)
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.)
Inozyme Pharma Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA3198957A1 publication Critical patent/CA3198957A1/en
Pending legal-status Critical Current

Links

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)
CA3198957A 2020-11-19 2021-11-19 Treatment of enpp1 deficiency and abcc6 deficiency Pending CA3198957A1 (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 (1)

Publication Number Publication Date
CA3198957A1 true CA3198957A1 (en) 2022-05-27

Family

ID=81709698

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3198957A Pending CA3198957A1 (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
AU2021383830A1 (en) 2023-06-22
JP2024500285A (ja) 2024-01-09
IL302954A (en) 2023-07-01
MX2023005836A (es) 2023-08-17

Similar Documents

Publication Publication Date Title
US11065306B2 (en) Methods for treating hypophosphatasia in children
US20260007725A1 (en) Alkaline phosphatase polypeptides and methods of use thereof
US20240181021A1 (en) Treatment of ENPP1 Deficiency and ABCC6 Deficiency
JP2023085277A (ja) 成人及び青年における低ホスファターゼ症（ｈｐｐ）を治療する方法
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
WO2025042995A1 (en) Treatment of enpp1 deficiency and abcc6 deficiency in children
CN116710122A (zh) Enpp1缺乏症和abcc6缺乏症的治疗
KR20260058843A (ko) 소아 enpp1 결핍증 및 abcc6 결핍증의 치료
HK40093022A (zh) Enpp1缺乏症和abcc6缺乏症的治疗
WO2025255052A1 (en) Treatment of enpp1 deficiency and abcc6 deficiency in infants
US20250073319A1 (en) Lyophilized enpp1 polypeptide formulations and uses thereof
BR112024019927B1 (pt) Uso de um composto para melhorar e/ou prevenir a hiperostose esquelética idiopática difusa (dish
HK40120234A (zh) 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
HK40112655A (zh) 冻干enpp1多肽制剂及其用途

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241115

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241115

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD

Year of fee payment: 4

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20251023

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20251023

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED

Effective date: 20251119

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT

Effective date: 20251119

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20251119

R11 Change to the name of applicant or owner or transfer of ownership requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R11-R127 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: TRANSFER RECORDAL REQUEST OR RESPONSE

Effective date: 20260112

R14 Transfer of ownership recorded

Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R14-R129 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: TRANSFER REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20260112

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260112

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20260127

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT

Effective date: 20260127

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20260127

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20260127

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260127

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260128