TW202229557A - Enpp1或enpp3之肝臟特異性生產 - Google Patents

Enpp1或enpp3之肝臟特異性生產 Download PDF

Info

Publication number
TW202229557A
TW202229557A TW110137622A TW110137622A TW202229557A TW 202229557 A TW202229557 A TW 202229557A TW 110137622 A TW110137622 A TW 110137622A TW 110137622 A TW110137622 A TW 110137622A TW 202229557 A TW202229557 A TW 202229557A
Authority
TW
Taiwan
Prior art keywords
leu
enpp1
ser
glu
pro
Prior art date
Application number
TW110137622A
Other languages
English (en)
Chinese (zh)
Inventor
史蒂芬 詹格斯
德米特里奧斯 布拉多克
Original Assignee
美商伊諾臻醫藥公司
耶魯大學
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 美商伊諾臻醫藥公司, 耶魯大學 filed Critical 美商伊諾臻醫藥公司
Publication of TW202229557A publication Critical patent/TW202229557A/zh

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • 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)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y306/00Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/01Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
    • C12Y306/01009Nucleotide diphosphatase (3.6.1.9), i.e. nucleotide-pyrophosphatase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/31Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Virology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
TW110137622A 2020-10-08 2021-10-08 Enpp1或enpp3之肝臟特異性生產 TW202229557A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202063089515P 2020-10-08 2020-10-08
US63/089,515 2020-10-08
US202163165650P 2021-03-24 2021-03-24
US63/165,650 2021-03-24
US202163248339P 2021-09-24 2021-09-24
US63/248,339 2021-09-24

Publications (1)

Publication Number Publication Date
TW202229557A true TW202229557A (zh) 2022-08-01

Family

ID=81126156

Family Applications (1)

Application Number Title Priority Date Filing Date
TW110137622A TW202229557A (zh) 2020-10-08 2021-10-08 Enpp1或enpp3之肝臟特異性生產

Country Status (7)

Country Link
US (1) US20240148907A1 (https=)
EP (1) EP4225927A4 (https=)
JP (1) JP2023548758A (https=)
AU (1) AU2021356692A1 (https=)
CA (1) CA3195071A1 (https=)
TW (1) TW202229557A (https=)
WO (1) WO2022076848A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023049864A1 (en) * 2021-09-24 2023-03-30 Inozyme Pharma, Inc. Lyophilized enpp1 polypeptide formulations and uses thereof
CN119242712A (zh) * 2024-09-27 2025-01-03 南京师范大学 特异性靶向骨骼肌细胞的携带miRNA-486基因的腺相关病毒载体、药物组合物

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CA2788682C (en) * 2010-02-05 2019-03-05 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
WO2014126965A2 (en) * 2013-02-13 2014-08-21 Yale University Compositions and methods for treating pathological calcification and ossification
KR20250078598A (ko) * 2015-05-19 2025-06-02 예일 유니버시티 병리학적 석회화 병태를 치료하기 위한 조성물, 및 이의 사용 방법
WO2017087936A1 (en) * 2015-11-20 2017-05-26 Yale University Compositions for treating ectopic calcification disorders, and methods using same
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
US20240148907A1 (en) 2024-05-09
AU2021356692A9 (en) 2024-06-13
EP4225927A1 (en) 2023-08-16
WO2022076848A1 (en) 2022-04-14
JP2023548758A (ja) 2023-11-21
AU2021356692A1 (en) 2023-05-04
CA3195071A1 (en) 2022-04-14
EP4225927A4 (en) 2025-05-07

Similar Documents

Publication Publication Date Title
US20230031809A1 (en) Treatment of diseases involving deficiency of enpp1 or enpp3
CN113227385B (zh) 核酸分子及其用于非病毒基因疗法的用途
KR102475799B1 (ko) 비-펩티드 결합을 함유하는 하이브리드 면역글로불린
JP2024032952A (ja) 異所性石灰化障害を治療するための組成物およびそれを使用する方法
US20240148907A1 (en) Liver specific production of enpp1 or enpp3
BRPI0612529A2 (pt) conjugados de anticorpo-medicamento de psma
KR20220095183A (ko) 바이러스 감염을 치료하기 위한 조성물 및 방법
KR20220157396A (ko) 면역 세포에서 아르기닌 수준을 조정하기 위한 방법 및 조성물
US20230330307A1 (en) Compositions and methods for inhibiting vascular smooth muscle cell proliferation
RU2853644C2 (ru) Лечение заболеваний, включающих дефицит enpp1 или enpp3
HK40063782B (zh) 与enpp1或enpp3缺乏有关的疾病的治疗
HK40063782A (en) Treatment of diseases involving deficiency of enpp1 or enpp3
TW202216777A (zh) 四面體抗體
CA3184349A1 (en) Compositions and methods for treating allograft vasculopathy, moyamoya disease, moyamoya syndrome and intimal proliferation
KR20220119056A (ko) 골관절염 치료 방법
US20250388925A1 (en) Wnt-modulating gene silencers as bone anabolic therapy for osteoporosis and critical-sized bone defect
RU2819144C2 (ru) Молекулы нуклеиновой кислоты и пути их применения
KR20240023127A (ko) 망막 장애
CN117813321A (zh) 视网膜病症
HK40093025A (zh) 用於治疗同种异体移植物血管病、烟雾病、烟雾病综合征和内膜增殖的组合物和方法