JP2024500285A - Ｅｎｐｐ１欠損症及びａｂｃｃ６欠損症の治療 - Google Patents

Ｅｎｐｐ１欠損症及びａｂｃｃ６欠損症の治療 Download PDF

Info

Publication number
JP2024500285A
JP2024500285A JP2023530251A JP2023530251A JP2024500285A JP 2024500285 A JP2024500285 A JP 2024500285A JP 2023530251 A JP2023530251 A JP 2023530251A JP 2023530251 A JP2023530251 A JP 2023530251A JP 2024500285 A JP2024500285 A JP 2024500285A
Authority
JP
Japan
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
JP2023530251A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024500285A5 (https=
Inventor
ウェルタス ペドロ
ヴェンカート デヴォラ
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
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 JP2024500285A publication Critical patent/JP2024500285A/ja
Publication of JP2024500285A5 publication Critical patent/JP2024500285A5/ja
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
    • 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
    • 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)
JP2023530251A 2020-11-19 2021-11-19 Ｅｎｐｐ１欠損症及びａｂｃｃ６欠損症の治療 Pending JP2024500285A (ja)

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
JP2024500285A true JP2024500285A (ja) 2024-01-09
JP2024500285A5 JP2024500285A5 (https=) 2024-11-26

Family

ID=81709698

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023530251A Pending JP2024500285A (ja) 2020-11-19 2021-11-19 Ｅｎｐｐ１欠損症及びａｂｃｃ６欠損症の治療

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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018500315A (ja) * 2014-12-19 2018-01-11 アレクシオン ファーマシューティカルズ インコーポレイテッドＡｌｅｘｉｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 組織石灰化の治療方法
JP2018516566A (ja) * 2015-05-19 2018-06-28 イエール ユニバーシティ 病的石灰化状態を治療するための組成物およびそれを使用する方法

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3126839A1 (en) * 2019-01-18 2020-07-23 Inozyme Pharma, Inc. Treatment of diseases involving deficiency of enpp1 or enpp3

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018500315A (ja) * 2014-12-19 2018-01-11 アレクシオン ファーマシューティカルズ インコーポレイテッドＡｌｅｘｉｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 組織石灰化の治療方法
JP2018516566A (ja) * 2015-05-19 2018-06-28 イエール ユニバーシティ 病的石灰化状態を治療するための組成物およびそれを使用する方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JOURNAL OF BONE AND MINERAL RESEARCH, vol. Vol. 35, Suppl. 1, JPN6025040539, 28 December 2020 (2020-12-28), pages 42, ISSN: 0005701290 *

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
CA3198957A1 (en) 2022-05-27
IL302954A (en) 2023-07-01
MX2023005836A (es) 2023-08-17

Similar Documents

Publication Publication Date Title
JP4874954B2 (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
DK2662448T3 (en) Bone-targeted alkaline phosphatase, kits and methods for its use
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
KR20260058843A (ko) 소아 enpp1 결핍증 및 abcc6 결핍증의 치료
HK40093022A (zh) Enpp1缺乏症和abcc6缺乏症的治疗
CN116710122A (zh) Enpp1缺乏症和abcc6缺乏症的治疗
WO2025255052A1 (en) Treatment of enpp1 deficiency and abcc6 deficiency in infants
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
BR112024019927B1 (pt) Uso de um composto para melhorar e/ou prevenir a hiperostose esquelética idiopática difusa (dish
US20250073319A1 (en) Lyophilized enpp1 polypeptide formulations and uses thereof
HK40112655A (zh) 冻干enpp1多肽制剂及其用途

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241115

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20241115

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20250929

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251006

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20251225

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20260305

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20260319

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260401

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20260323