DK2797613T3 - Vektorer, der koder for rod-derived cone viability-faktor - Google Patents

Vektorer, der koder for rod-derived cone viability-faktor Download PDF

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Publication number
DK2797613T3
DK2797613T3 DK12843778.7T DK12843778T DK2797613T3 DK 2797613 T3 DK2797613 T3 DK 2797613T3 DK 12843778 T DK12843778 T DK 12843778T DK 2797613 T3 DK2797613 T3 DK 2797613T3
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Prior art keywords
rod
vectors coding
viability factor
cone viability
derived cone
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DK12843778.7T
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Danish (da)
English (en)
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Tianci Luo
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Wellstat Ophthalmics Corp
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    • 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
    • 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
    • C07K14/46—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • 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
    • A—HUMAN NECESSITIES
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    • 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
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    • 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
    • A61P25/16—Anti-Parkinson drugs
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
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    • A61P27/00—Drugs for disorders of the senses
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P27/02—Ophthalmic agents
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    • A61P27/06—Antiglaucoma agents or miotics
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    • A61P27/08—Mydriatics or cycloplegics
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    • A—HUMAN NECESSITIES
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    • A61P27/14—Decongestants or antiallergics
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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
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0051—Oxidoreductases (1.) acting on a sulfur group of donors (1.8)
    • 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
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
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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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    • C12N2830/00—Vector systems having a special element relevant for transcription
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DK12843778.7T 2011-10-27 2012-10-26 Vektorer, der koder for rod-derived cone viability-faktor DK2797613T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161552155P 2011-10-27 2011-10-27
PCT/US2012/062106 WO2013063383A2 (en) 2011-10-27 2012-10-26 Vectors encoding rod-derived cone viability factor

Publications (1)

Publication Number Publication Date
DK2797613T3 true DK2797613T3 (da) 2020-03-02

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DK12843778.7T DK2797613T3 (da) 2011-10-27 2012-10-26 Vektorer, der koder for rod-derived cone viability-faktor

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US (2) US9265813B2 (cg-RX-API-DMAC7.html)
EP (1) EP2797613B1 (cg-RX-API-DMAC7.html)
JP (1) JP6293664B2 (cg-RX-API-DMAC7.html)
KR (1) KR102000141B1 (cg-RX-API-DMAC7.html)
CN (2) CN104321069A (cg-RX-API-DMAC7.html)
AU (1) AU2012321102C1 (cg-RX-API-DMAC7.html)
BR (1) BR112014010091B1 (cg-RX-API-DMAC7.html)
CA (1) CA2853379C (cg-RX-API-DMAC7.html)
DK (1) DK2797613T3 (cg-RX-API-DMAC7.html)
ES (1) ES2774779T3 (cg-RX-API-DMAC7.html)
MX (1) MX354516B (cg-RX-API-DMAC7.html)
PL (1) PL2797613T3 (cg-RX-API-DMAC7.html)
RU (1) RU2664673C2 (cg-RX-API-DMAC7.html)
WO (1) WO2013063383A2 (cg-RX-API-DMAC7.html)

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US10004780B2 (en) * 2012-10-17 2018-06-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of age-related macular degeneration (AMD)
FI3851537T3 (fi) 2014-04-25 2024-03-21 Genethon Hyperbilirubinemian hoito
JP6684343B2 (ja) * 2015-05-21 2020-04-22 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ニューロン生存因子の相乗組み合わせ及びその使用
GB201516066D0 (en) * 2015-09-10 2015-10-28 Young & Co Llp D Treatment of retinitis pigmentosa
JP7061067B2 (ja) 2015-12-14 2022-04-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クリグラー・ナジャー症候群の処置のための組成物
US10857240B2 (en) 2016-01-05 2020-12-08 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of ocular disorders and blinding diseases
CN106148528B (zh) * 2016-07-11 2019-10-01 赵晨 一种遗传性Usher综合征的致病突变及其检测试剂
US10946063B2 (en) * 2016-10-11 2021-03-16 Welltat Ophthalmics Corporation Fusion protein between short form rod-derived cone viability factor and a hydrophilic peptide
KR102776626B1 (ko) * 2017-10-16 2025-03-04 비게네론 게엠베하 Aav 벡터
BR112020012471A2 (pt) * 2017-12-22 2020-11-24 Sparingvision construtos compreendendo fatores de viabilidade neuronal e usos dos mesmos
JP7213891B2 (ja) * 2018-01-30 2023-01-27 ユニベルシテイト ゲント 白内障を処置するのに使用するための組成物
CN108504685A (zh) * 2018-03-27 2018-09-07 宜明细胞生物科技有限公司 一种利用CRISPR/Cas9系统同源重组修复IL-2RG缺陷基因的方法
SG11202102140WA (en) * 2018-09-07 2021-04-29 Univ Southern California Cryopreservation of cell-seeded substrates and related methods
KR20210092755A (ko) * 2018-11-14 2021-07-26 리젠엑스바이오 인크. 신경원성 세로이드 리포푸신증에 대한 유전자 요법
PE20211342A1 (es) * 2018-12-19 2021-07-26 Versameb Ag Acido ribonucleico (arn) que codifica para una proteina
CN114651003A (zh) 2019-09-10 2022-06-21 黑曜石疗法公司 用于可调调节的ca2-il15融合蛋白
EP4074828A4 (en) * 2019-12-09 2023-12-27 Chigenovo Co., Ltd. Uses of cyp4v2 and rdcvf in preparation of drugs
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
KR20230092880A (ko) * 2020-08-28 2023-06-26 조인트 스탁 컴퍼니 "바이오케드" AAV5-기반의 SARS-CoV-2 백신
WO2024206928A1 (en) 2023-03-30 2024-10-03 Pharma Cinq, Llc VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IgK SIGNAL SEQUENCE

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JP2015501156A (ja) 2015-01-15
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EP2797613A2 (en) 2014-11-05
EP2797613A4 (en) 2015-09-09
PL2797613T3 (pl) 2020-06-15
US10040835B2 (en) 2018-08-07
MX354516B (es) 2018-03-08
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AU2012321102C1 (en) 2016-11-10
BR112014010091A2 (pt) 2020-10-27
US9265813B2 (en) 2016-02-23
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WO2013063383A3 (en) 2013-06-20
CA2853379A1 (en) 2013-05-02
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US20140328821A1 (en) 2014-11-06
EP2797613B1 (en) 2019-12-04
CN104321069A (zh) 2015-01-28
AU2012321102B2 (en) 2016-07-14
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