JP7028802B2 - 短鎖型桿体由来錐体生存因子及び親水性ペプチド間の融合タンパク質 - Google Patents

短鎖型桿体由来錐体生存因子及び親水性ペプチド間の融合タンパク質 Download PDF

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JP7028802B2
JP7028802B2 JP2018563544A JP2018563544A JP7028802B2 JP 7028802 B2 JP7028802 B2 JP 7028802B2 JP 2018563544 A JP2018563544 A JP 2018563544A JP 2018563544 A JP2018563544 A JP 2018563544A JP 7028802 B2 JP7028802 B2 JP 7028802B2
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fusion protein
sequence
peptide sequence
rdcvf
vector
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JP2019532616A (ja
JP2019532616A5 (enExample
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ティアンチ ルオ
ジュン ジャン
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Wellstat Opthalmics Corp
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/643—Albumins, e.g. HSA, BSA, ovalbumin or a Keyhole Limpet Hemocyanin [KHL]
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K14/76—Albumins
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0036—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on NADH or NADPH (1.6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y108/00—Oxidoreductases acting on sulfur groups as donors (1.8)
    • C12Y108/01—Oxidoreductases acting on sulfur groups as donors (1.8) with NAD+ or NADP+ as acceptor (1.8.1)
    • C12Y108/01008—Protein-disulfide reductase (1.8.1.8), i.e. thioredoxin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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JP2018563544A 2016-10-11 2017-10-11 短鎖型桿体由来錐体生存因子及び親水性ペプチド間の融合タンパク質 Active JP7028802B2 (ja)

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Application Number Priority Date Filing Date Title
US201662406552P 2016-10-11 2016-10-11
US62/406,552 2016-10-11
PCT/US2017/056030 WO2018071465A1 (en) 2016-10-11 2017-10-11 Fusion protein between short form rod-derived cone viability factor and a hydrophilic peptide

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JP2019532616A JP2019532616A (ja) 2019-11-14
JP2019532616A5 JP2019532616A5 (enExample) 2020-11-19
JP7028802B2 true JP7028802B2 (ja) 2022-03-02

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US (3) US10946063B2 (enExample)
EP (1) EP3526238A4 (enExample)
JP (1) JP7028802B2 (enExample)
KR (2) KR20230156440A (enExample)
CN (1) CN109415423A (enExample)
AU (1) AU2017344059B2 (enExample)
BR (1) BR112018076674A2 (enExample)
CA (1) CA3025977A1 (enExample)
IL (1) IL263990A (enExample)
MX (1) MX2018015596A (enExample)
NZ (1) NZ748634A (enExample)
WO (1) WO2018071465A1 (enExample)
ZA (1) ZA201808041B (enExample)

Families Citing this family (4)

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Publication number Priority date Publication date Assignee Title
CN113528575B (zh) * 2018-08-07 2022-06-21 康码（上海）生物科技有限公司 信号肽相关序列及其在蛋白质合成中的应用
US20230146121A1 (en) * 2019-12-09 2023-05-11 Chigenovo Co., Ltd. Use of cyp4v2 and rdcvf in the manufacture of medicament
CN111733174B (zh) * 2020-08-07 2021-02-09 北京大学第三医院（北京大学第三临床医学院） 一种分离的核酸分子及其用途
WO2024206928A1 (en) * 2023-03-30 2024-10-03 Pharma Cinq, Llc VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IgK SIGNAL SEQUENCE

Citations (4)

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WO2009146183A1 (en) 2008-04-15 2009-12-03 Genzyme Corporation Methods to produce rod-derived cone viability factor (rdcvf)
JP2010529958A (ja) 2007-06-05 2010-09-02 アンスティテュ、ナショナル、ド、ラ、サント、エ、ド、ラ、ルシェルシュ、メディカル（アンセルム） ニューロン生存因子およびその使用
JP2014516358A (ja) 2011-04-12 2014-07-10 ガンガゲン インコーポレーティッド キメラ抗菌ポリペプチド
JP2015501156A (ja) 2011-10-27 2015-01-15 ウェルスタット オフサルミクス コーポレイション 桿体由来錐体生存因子をコードするベクター

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EP0943003B1 (en) 1996-12-02 2004-11-17 Valentis Inc. Insulin-like growth factor i (igf-i) expression system and methods of use
FR2823221B1 (fr) 2001-04-06 2004-04-02 Univ Pasteur Sequences associees a la degenerescence retinienne et applications
CA2484556A1 (en) * 2001-12-21 2003-07-24 Human Genome Sciences, Inc. Albumin fusion proteins
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DE10260805A1 (de) 2002-12-23 2004-07-22 Geneart Gmbh Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins
FR2870241B1 (fr) 2004-05-13 2015-02-27 Novartis Ag Facteur de viabilite des cones derive des batonnets ou rdcvf et applications
WO2010029130A1 (en) 2008-09-10 2010-03-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Neuronal viability factor and use thereof
US20120088819A1 (en) 2008-10-31 2012-04-12 Makoto Inoue Method for enhancing expression of recombinant protein
IN2015DN01115A (enExample) 2012-07-13 2015-06-26 Zymeworks Inc
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WO2017120294A1 (en) 2016-01-05 2017-07-13 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of ocular disorders and blinding diseases

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JP2010529958A (ja) 2007-06-05 2010-09-02 アンスティテュ、ナショナル、ド、ラ、サント、エ、ド、ラ、ルシェルシュ、メディカル（アンセルム） ニューロン生存因子およびその使用
WO2009146183A1 (en) 2008-04-15 2009-12-03 Genzyme Corporation Methods to produce rod-derived cone viability factor (rdcvf)
JP2014516358A (ja) 2011-04-12 2014-07-10 ガンガゲン インコーポレーティッド キメラ抗菌ポリペプチド
JP2015501156A (ja) 2011-10-27 2015-01-15 ウェルスタット オフサルミクス コーポレイション 桿体由来錐体生存因子をコードするベクター

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US10946063B2 (en) 2021-03-16
RU2018144780A (ru) 2020-11-17
CN109415423A (zh) 2019-03-01
BR112018076674A2 (pt) 2019-04-02
US20240374683A1 (en) 2024-11-14
JP2019532616A (ja) 2019-11-14
EP3526238A4 (en) 2020-04-29
NZ748634A (en) 2025-11-28
US20190151410A1 (en) 2019-05-23
ZA201808041B (en) 2019-09-25
KR20190058388A (ko) 2019-05-29
CA3025977A1 (en) 2018-04-19
WO2018071465A1 (en) 2018-04-19
AU2017344059A1 (en) 2018-12-13
RU2018144780A3 (enExample) 2021-02-02
KR20230156440A (ko) 2023-11-14
US12076367B2 (en) 2024-09-03
AU2017344059B2 (en) 2021-12-23
MX2018015596A (es) 2019-03-14
US20210162005A1 (en) 2021-06-03
EP3526238A1 (en) 2019-08-21
IL263990A (en) 2019-01-31

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