KR20210142599A - Enpp1 또는 enpp3 결핍을 수반하는 질환의 치료 - Google Patents

Enpp1 또는 enpp3 결핍을 수반하는 질환의 치료 Download PDF

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KR20210142599A
KR20210142599A KR1020217025330A KR20217025330A KR20210142599A KR 20210142599 A KR20210142599 A KR 20210142599A KR 1020217025330 A KR1020217025330 A KR 1020217025330A KR 20217025330 A KR20217025330 A KR 20217025330A KR 20210142599 A KR20210142599 A KR 20210142599A
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enpp1
viral vector
enpp3
vector
signal peptide
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Korean (ko)
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스티븐 정글스
데메트리어스 브라독
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이노자임 파마, 인코포레이티드
예일 유니버시티
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/02—Breeding vertebrates
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/14011—Parvoviridae
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KR1020217025330A 2019-01-18 2020-01-20 Enpp1 또는 enpp3 결핍을 수반하는 질환의 치료 Ceased KR20210142599A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962794450P 2019-01-18 2019-01-18
US62/794,450 2019-01-18
US201962821692P 2019-03-21 2019-03-21
US62/821,692 2019-03-21
US201962877044P 2019-07-22 2019-07-22
US62/877,044 2019-07-22
PCT/US2020/014296 WO2020150716A1 (en) 2019-01-18 2020-01-20 Treatment of diseases involving deficiency of enpp1 or enpp3

Publications (1)

Publication Number Publication Date
KR20210142599A true KR20210142599A (ko) 2021-11-25

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KR1020217025330A Ceased KR20210142599A (ko) 2019-01-18 2020-01-20 Enpp1 또는 enpp3 결핍을 수반하는 질환의 치료

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US (2) US20210187067A1 (https=)
EP (1) EP3911153A4 (https=)
JP (2) JP7724156B2 (https=)
KR (1) KR20210142599A (https=)
CN (1) CN113631033B (https=)
AU (1) AU2020207967A1 (https=)
BR (1) BR112021013941A2 (https=)
CA (1) CA3126839A1 (https=)
IL (1) IL284699A (https=)
MX (1) MX2021008503A (https=)
WO (1) WO2020150716A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022076848A1 (en) * 2020-10-08 2022-04-14 Inozyme Pharma, Inc. Liver specific production of enpp1 or enpp3
AU2021383830A1 (en) * 2020-11-19 2023-06-22 Biomarin Pharmaceutical Inc. Treatment of enpp1 deficiency and abcc6 deficiency
WO2023049864A1 (en) * 2021-09-24 2023-03-30 Inozyme Pharma, Inc. Lyophilized enpp1 polypeptide formulations and uses thereof
KR20240167914A (ko) * 2022-04-04 2024-11-28 이노자임 파마, 인코포레이티드 Enpp1 결핍증 및 abcc6 결핍증의 치료
WO2025213139A1 (en) * 2024-04-04 2025-10-09 The General Hospital Corporation Enpp1 gene therapy for the treatment of vascular disease

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6894022B1 (en) * 1998-08-27 2005-05-17 Eidgenossische Technische Hochschule Zurich Growth factor modified protein matrices for tissue engineering
CA2462653C (en) * 2001-11-07 2016-06-07 Aya Jakobovits Nucleic acid and corresponding protein entitled 161p2f10b useful in treatment and detection of cancer
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
US8168586B1 (en) * 2007-11-21 2012-05-01 Celera Corporation Cancer targets and uses thereof
WO2010037408A1 (en) * 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
EP3470089A1 (en) * 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
CA2960912A1 (en) * 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3234116B1 (en) * 2014-12-19 2021-09-08 Alexion Pharmaceuticals, Inc. Methods of treating tissue calcification
KR20250078598A (ko) * 2015-05-19 2025-06-02 예일 유니버시티 병리학적 석회화 병태를 치료하기 위한 조성물, 및 이의 사용 방법
BR112019002355A2 (pt) * 2016-08-05 2019-07-16 Yale University composições e métodos para prevenção de acidente vascular cerebral em pacientes pediátricos com anemia falciforme
EP3844280A4 (en) * 2018-08-31 2022-09-14 Yale University ENPP1 POLYPEPTIDES AND METHODS OF USE

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Publication number Publication date
IL284699A (en) 2021-08-31
BR112021013941A2 (pt) 2021-09-21
CA3126839A1 (en) 2020-07-23
MX2021008503A (es) 2021-10-13
CN113631033A (zh) 2021-11-09
US20210187067A1 (en) 2021-06-24
EP3911153A4 (en) 2022-12-21
US20230031809A1 (en) 2023-02-02
CN113631033B (zh) 2024-10-15
JP2022517435A (ja) 2022-03-08
WO2020150716A1 (en) 2020-07-23
JP2025084821A (ja) 2025-06-03
EP3911153A1 (en) 2021-11-24
JP7724156B2 (ja) 2025-08-15
AU2020207967A1 (en) 2021-07-29

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