ES2983910T3 - Composiciones para tratar afecciones patológicas de calcificación y métodos que usan las mismas - Google Patents

Composiciones para tratar afecciones patológicas de calcificación y métodos que usan las mismas Download PDF

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Publication number
ES2983910T3
ES2983910T3 ES16797290T ES16797290T ES2983910T3 ES 2983910 T3 ES2983910 T3 ES 2983910T3 ES 16797290 T ES16797290 T ES 16797290T ES 16797290 T ES16797290 T ES 16797290T ES 2983910 T3 ES2983910 T3 ES 2983910T3
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seq
disclosure
polypeptide
npp1
protein
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Spanish (es)
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Demetrios Braddock
Ronald Albright
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Yale University
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Yale University
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1741—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals alpha-Glycoproteins
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0271—Chimeric vertebrates, e.g. comprising exogenous cells
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/02—Peptides of undefined number of amino acids; Derivatives thereof
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
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    • C12N15/52—Genes encoding for enzymes or proenzymes
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
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    • 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
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    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • 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
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    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
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    • A01K2207/00—Modified animals
    • A01K2207/20—Animals treated with compounds which are neither proteins nor nucleic acids
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • A01K2267/0387—Animal model for diseases of the immune system
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
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    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
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    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
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    • C07K2319/00—Fusion polypeptide
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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ES16797290T 2015-05-19 2016-05-19 Composiciones para tratar afecciones patológicas de calcificación y métodos que usan las mismas Active ES2983910T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562163500P 2015-05-19 2015-05-19
PCT/US2016/033236 WO2016187408A1 (en) 2015-05-19 2016-05-19 Compositions for treating pathological calcification conditions, and methods using same

Publications (1)

Publication Number Publication Date
ES2983910T3 true ES2983910T3 (es) 2024-10-28

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ES16797290T Active ES2983910T3 (es) 2015-05-19 2016-05-19 Composiciones para tratar afecciones patológicas de calcificación y métodos que usan las mismas

Country Status (11)

Country Link
US (10) US10583170B2 (https=)
EP (2) EP4414028A3 (https=)
JP (4) JP6995627B2 (https=)
KR (2) KR20250078598A (https=)
AU (3) AU2016264364B2 (https=)
CA (2) CA2984947A1 (https=)
ES (1) ES2983910T3 (https=)
MX (2) MX391074B (https=)
PL (1) PL3298140T3 (https=)
RU (2) RU2757417C2 (https=)
WO (1) WO2016187408A1 (https=)

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WO2017087936A1 (en) 2015-11-20 2017-05-26 Yale University Compositions for treating ectopic calcification disorders, and methods using same
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US11696954B2 (en) 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
EP3687565A1 (en) * 2017-09-27 2020-08-05 Alexion Pharmaceuticals, Inc. Methods of improving cardiovascular function and treating cardiovascular disease using a recombinant ectonucleotide pyrophosphatase phosphodiesterase (npp1)
JP2021523139A (ja) 2018-05-08 2021-09-02 イエール ユニバーシティ 腎石症の進行を遅らせるための組成物および方法
EP3844280A4 (en) * 2018-08-31 2022-09-14 Yale University ENPP1 POLYPEPTIDES AND METHODS OF USE
KR20210142599A (ko) * 2019-01-18 2021-11-25 이노자임 파마, 인코포레이티드 Enpp1 또는 enpp3 결핍을 수반하는 질환의 치료
CN113613635A (zh) 2019-01-24 2021-11-05 西北大学 使用脂质缀合的核心支架的高密度脂蛋白模拟纳米颗粒
BR112021020037A2 (pt) * 2019-04-05 2021-12-07 Univ Yale Polipeptídeos enpp1 e métodos para o uso dos mesmos
CN116406281A (zh) * 2020-05-27 2023-07-07 依诺兹梅制药公司 用于治疗外周动脉疾病的组合物和方法
IL298853A (en) 2020-06-09 2023-02-01 Inozyme Pharma Inc Soluble enpp1 or enpp3 proteins and uses thereof
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
IL326528A (en) * 2023-08-22 2026-04-01 Inozyme Pharma Inc Treatment of ENPP1 deficiency and ABCC6 deficiency in children
WO2025255052A1 (en) * 2024-06-03 2025-12-11 Inozyme Pharma, Inc. Treatment of enpp1 deficiency and abcc6 deficiency in infants

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