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
    • 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
    • A01K67/0271—Chimeric vertebrates, e.g. comprising exogenous cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/02—Peptides of undefined number of amino acids; Derivatives thereof
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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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    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C07—ORGANIC CHEMISTRY
    • 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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    • C12N15/00—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
    • C12N15/52—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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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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    • 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
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    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
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    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • 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
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0387—Animal model for diseases of the immune system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • 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
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
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    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
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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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  • Pharmacology & Pharmacy (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Urology & Nephrology (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

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Country Link
US (10) US10064917B2 (cg-RX-API-DMAC7.html)
EP (2) EP3298140B1 (cg-RX-API-DMAC7.html)
JP (3) JP6995627B2 (cg-RX-API-DMAC7.html)
KR (2) KR102811896B1 (cg-RX-API-DMAC7.html)
AU (3) AU2016264364B2 (cg-RX-API-DMAC7.html)
CA (2) CA3176123A1 (cg-RX-API-DMAC7.html)
ES (1) ES2983910T3 (cg-RX-API-DMAC7.html)
MX (2) MX391074B (cg-RX-API-DMAC7.html)
PL (1) PL3298140T3 (cg-RX-API-DMAC7.html)
RU (2) RU2021129201A (cg-RX-API-DMAC7.html)
WO (1) WO2016187408A1 (cg-RX-API-DMAC7.html)

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EP2956163B1 (en) 2013-02-13 2024-11-27 Yale University Compositions for treating pathological calcification and ossification
WO2015187966A1 (en) 2014-06-04 2015-12-10 Aurasense Therapeutics, Llc Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
BR112017012928A2 (pt) * 2014-12-19 2018-05-15 Alexion Pharma Inc uso de um ectonucleotídeo pirofosfatase fosfodiesterase (npp1); uso de uma composição farmacêutica; e uso de um snpp1 humano recombinante isolado, fragmento ou proteína de fusão deste
CA3176123A1 (en) * 2015-05-19 2016-11-24 Yale University Compositions for treating pathological calcification conditions, and methods using same
JP2018537093A (ja) 2015-11-20 2018-12-20 イエール ユニバーシティ 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
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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
WO2018201090A1 (en) 2017-04-28 2018-11-01 Exicure, Inc. Synthesis of spherical nucleic acids using lipophilic moieties
WO2019067502A1 (en) * 2017-09-27 2019-04-04 Alexion Pharmaceuticals, Inc. METHODS FOR IMPROVING CARDIOVASCULAR FUNCTION AND TREATING CARDIOVASCULAR DISEASE USING RECOMBINANT ECTO-NUCLEOTIDE PYROPHOSPHATASE / PHOSPHODIESTERASE
CA3099266A1 (en) 2018-05-08 2019-11-14 Yale University Compositions and methods for reducing progression of nephrolithiasis
CN112930397B (zh) * 2018-08-31 2025-05-09 耶鲁大学 Enpp1多肽及其使用方法
BR112021013941A2 (pt) * 2019-01-18 2021-09-21 Inozyme Pharma, Inc. Polinucleotídeo recombinante, vetor viral, método para obter um vetor viral recombinante, método para fornecer proteína enpp1 ou enpp3 a um mamífero, composição farmacêutica, método para evitar ou reduzir a progressão de uma doença em um mamífero em necessidade do mesmo, célula, método para tratar ou evitar uma doença ou distúrbio de calcificação patológica ou ossificação patológica em um indivíduo em necessidade do mesmo, método para tratar um indivíduo com deficiência de proteína enpp1, método para produzir um vetor viral recombinante e vetor viral recombinante
MX2021008940A (es) 2019-01-24 2021-11-04 Univ Northwestern Nanoparticulas mimeticas de lipoproteinas de alta densidad usando andamios de nucleos conjugados con lipidos.
MX2021012197A (es) * 2019-04-05 2022-01-06 Univ Yale Polipeptidos de ectonucleotido pirofosfatasa (enpp1) y metodos de uso de los mismos.
KR20230048480A (ko) * 2020-05-27 2023-04-11 이노자임 파마, 인코포레이티드 말초 동맥 질환을 치료하기 위한 조성물 및 방법
EP4162036A1 (en) 2020-06-09 2023-04-12 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
MX2023005836A (es) * 2020-11-19 2023-08-17 Inozyme Pharma Inc Tratamiento para la deficiencia de enpp1 y la deficiencia de abcc6.
AU2022353101A1 (en) * 2021-09-24 2024-05-09 Biomarin Pharmaceutical Inc. Lyophilized enpp1 polypeptide formulations and uses thereof
WO2025042995A1 (en) * 2023-08-22 2025-02-27 Inozyme Pharma, Inc. Treatment of enpp1 deficiency and abcc6 deficiency in children

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