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
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    • 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
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • 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
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    • A61P27/14—Decongestants or antiallergics
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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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
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    • 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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    • 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
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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 (enExample)
EP (1) EP2797613B1 (enExample)
JP (1) JP6293664B2 (enExample)
KR (1) KR102000141B1 (enExample)
CN (2) CN104321069A (enExample)
AU (1) AU2012321102C1 (enExample)
BR (1) BR112014010091B1 (enExample)
CA (1) CA2853379C (enExample)
DK (1) DK2797613T3 (enExample)
ES (1) ES2774779T3 (enExample)
MX (1) MX354516B (enExample)
PL (1) PL2797613T3 (enExample)
RU (1) RU2664673C2 (enExample)
WO (1) WO2013063383A2 (enExample)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2027889A1 (en) * 2007-06-05 2009-02-25 Institut National De La Sante Et De La Recherche Medicale (Inserm) New neuronal viability factor and use thereof
WO2014060517A1 (en) * 2012-10-17 2014-04-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of age-related macular degeneration (amd)
DK3546585T3 (da) * 2014-04-25 2025-11-03 Genethon Nukleinsyrer med en modificeret intron til brug i behandlingen af hyperbilirubinæmi
WO2016185242A1 (en) 2015-05-21 2016-11-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Synergistic combination of neuronal viability factors and uses thereof
GB201516066D0 (en) * 2015-09-10 2015-10-28 Young & Co Llp D Treatment of retinitis pigmentosa
US11241506B2 (en) 2015-12-14 2022-02-08 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
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
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
CN111601884A (zh) * 2017-10-16 2020-08-28 维格内罗有限责任公司 Aav载体
SG11202006796RA (en) * 2017-12-22 2020-08-28 Sparingvision Constructs comprising neuronal viability factors and uses thereof
ES2917628T3 (es) * 2018-01-30 2022-07-11 Univ Gent Composiciones a utilizar para tratar cataratas
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
US11825839B2 (en) * 2018-09-07 2023-11-28 University Of Southern California Cryopreservation of cell-seeded substrates and related methods
BR112021009370A2 (pt) * 2018-11-14 2021-08-17 Regenxbio Inc. método de tratamento da doença de batten cln2, composição farmacêutica e kit
KR20210105382A (ko) * 2018-12-19 2021-08-26 베르사멥 아게 단백질을 코딩하는 rna
WO2021050789A1 (en) 2019-09-10 2021-03-18 Obsidian Therapeutics, Inc. Ca2-il15 fusion proteins for tunable regulation
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
WO2022045935A1 (en) * 2020-08-28 2022-03-03 Joint Stock Company "Biocad" Aav5-based vaccine against sars-cov-2
WO2024206928A1 (en) 2023-03-30 2024-10-03 Pharma Cinq, Llc VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IgK SIGNAL SEQUENCE

Family Cites Families (66)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5840293A (en) 1988-11-16 1998-11-24 Advanced Polymer Systems, Inc. Ionic beads for controlled release and adsorption
US5225347A (en) 1989-09-25 1993-07-06 Innovir Laboratories, Inc. Therapeutic ribozyme compositions and expression vectors
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DK0585368T3 (da) 1991-04-25 1998-03-16 Univ Brown Res Found Implanterbart biokompatibelt immunisolatorisk vehikel til afgivelse af udvalgte terapeutiske produkter
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US6040172A (en) 1992-08-14 2000-03-21 The Rockefeller University Defective DNA viral vector comprising a neural tissue-specific promoter for in vivo expression of a gene
CA2146747C (en) 1992-10-09 2006-12-19 Brian A. Naughton Liver reserve cells
GB9223084D0 (en) 1992-11-04 1992-12-16 Imp Cancer Res Tech Compounds to target cells
US5908623A (en) 1993-08-12 1999-06-01 Cytotherapeutics, Inc. Compositions and methods for the delivery of biologically active molecules using genetically altered cells contained in biocompatible immunoisolatory capsules
US5552311A (en) 1993-09-14 1996-09-03 University Of Alabama At Birmingham Research Foundation Purine nucleoside phosphorylase gene therapy for human malignancy
TW442569B (en) 1993-10-25 2001-06-23 Canji Inc Recombinant adenoviral vector
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5763416A (en) 1994-02-18 1998-06-09 The Regent Of The University Of Michigan Gene transfer into bone cells and tissues
US5942496A (en) 1994-02-18 1999-08-24 The Regent Of The University Of Michigan Methods and compositions for multiple gene transfer into bone cells
FR2716459B1 (fr) 1994-02-22 1996-05-10 Univ Paris Curie Système hôte-vecteur utilisable en thérapie génique.
WO1995024929A2 (en) 1994-03-15 1995-09-21 Brown University Research Foundation Polymeric gene delivery system
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
WO1995034671A1 (en) 1994-06-10 1995-12-21 Genvec, Inc. Complementary adenoviral vector systems and cell lines
CA2203809C (en) 1994-10-28 2008-06-03 James M. Wilson Recombinant adenovirus and methods of use thereof
US5843742A (en) 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
US5880102A (en) 1995-01-17 1999-03-09 Duke University Adenoviral vector system
US5707618A (en) 1995-03-24 1998-01-13 Genzyme Corporation Adenovirus vectors for gene therapy
DE19514541C2 (de) 1995-04-20 1998-01-15 Daimler Benz Ag Gaspedal mit Reibkörper
US5773289A (en) 1995-06-06 1998-06-30 University Of Pittsburgh AAV directed targeted integration
US5869040A (en) 1995-06-07 1999-02-09 Biogen, Inc Gene therapy methods and compositions
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
DE19541450C2 (de) 1995-11-07 1997-10-02 Gsf Forschungszentrum Umwelt Genkonstrukt und dessen Verwendung
US5830727A (en) 1995-11-18 1998-11-03 Human Gene Therapy Research Institute Herpes simplex virus amplicon mini-vector gene transfer system
US20030040467A1 (en) 1998-06-15 2003-02-27 Mary Ann Pelleymounter Ig/ob fusions and uses thereof.
US6299895B1 (en) 1997-03-24 2001-10-09 Neurotech S.A. Device and method for treating ophthalmic diseases
US6878544B2 (en) 1996-04-19 2005-04-12 Neurotech Sa Retinal cell lines with extended life-span and their applications
US6054142A (en) 1996-08-01 2000-04-25 Cyto Therapeutics, Inc. Biocompatible devices with foam scaffolds
US5994132A (en) 1996-10-23 1999-11-30 University Of Michigan Adenovirus vectors
EP0943003B1 (en) * 1996-12-02 2004-11-17 Valentis Inc. Insulin-like growth factor i (igf-i) expression system and methods of use
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6303136B1 (en) 1998-04-13 2001-10-16 Neurotech S.A. Cells or tissue attached to a non-degradable filamentous matrix encapsulated by a semi-permeable membrane
US5981225A (en) 1998-04-16 1999-11-09 Baylor College Of Medicine Gene transfer vector, recombinant adenovirus particles containing the same, method for producing the same and method of use of the same
US5994134A (en) 1998-05-04 1999-11-30 Canji, Inc. Viral production process
EP0974660A1 (en) 1998-06-19 2000-01-26 Stichting Instituut voor Dierhouderij en Diergezondheid (ID-DLO) Newcastle disease virus infectious clones, vaccines and diagnostic assays
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
US6146642A (en) 1998-09-14 2000-11-14 Mount Sinai School Of Medicine, Of The City University Of New York Recombinant new castle disease virus RNA expression systems and vaccines
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6303362B1 (en) 1998-11-19 2001-10-16 The Board Of Trustees Of The Leland Stanford Junior University Adenoviral vector and methods for making and using the same
US6361771B1 (en) 1999-04-06 2002-03-26 Neurotech S.A. ARPE-19 as a platform cell line for encapsulated cell-based delivery
US6864085B2 (en) 1999-12-14 2005-03-08 Novartis Ag Bovine immunodeficiency virus (BIV) based vectors
US7098030B2 (en) 1999-12-31 2006-08-29 Mirus Bio Corporation Polyampholytes for delivering polyions to a cell
IL140844A0 (en) 2001-01-10 2002-02-10 Polygene Ltd Cationic polysaccharide compositions
FR2823221B1 (fr) 2001-04-06 2004-04-02 Univ Pasteur Sequences associees a la degenerescence retinienne et applications
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
AU2003225544A1 (en) * 2002-02-04 2003-09-02 Novartis Ag Recombinant bovine immunodeficiency virus based gene transfer system
ATE536405T1 (de) 2002-07-25 2011-12-15 Scripps Research Inst Hämatopoetische stammzellen und verfahren zur behandlung von neovaskulären augenerkrankungen damit
DE10260805A1 (de) 2002-12-23 2004-07-22 Geneart Gmbh Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins
EP1624758A4 (en) * 2003-05-02 2007-03-07 Scripps Research Inst HEMOPOETIC STEM CELLS AND METHOD FOR THE TREATMENT OF EYE-RELATED VASCULAR DISEASES USING THESE STEM CELLS
FR2870241B1 (fr) 2004-05-13 2015-02-27 Novartis Ag Facteur de viabilite des cones derive des batonnets ou rdcvf et applications
JP5247468B2 (ja) 2005-12-30 2013-07-24 ニューロテック ユーエスエー， インコーポレイテッド 生物活性分子の送達のための微粒子化デバイスおよびその使用方法
KR20090122465A (ko) 2007-03-01 2009-11-30 어드벤스드 비젼 테라피스, 인코포레이티드 염증성 질환의 치료
EP2027889A1 (en) * 2007-06-05 2009-02-25 Institut National De La Sante Et De La Recherche Medicale (Inserm) New neuronal viability factor and use thereof
JP5710265B2 (ja) * 2007-12-06 2015-04-30 アンセルム（アンスチチュ 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） 網膜変性疾患の治療のための栄養因子
EP2281047B1 (en) 2008-04-15 2020-04-08 Genzyme Corporation Methods to produce rod-derived cone viability factor (rdcvf)
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
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
DE102009016934A1 (de) 2009-04-08 2010-10-14 Daimler Ag Brennstoffzelle, Brennstoffzellenstapel und Verfahren zum Abdichten einer Brennstoffzelle
JP2013507373A (ja) 2009-10-08 2013-03-04 ニューロテック ユーエスエー， インコーポレイテッド 被包された細胞ベースの送達系におけるｐｅｄｆの使用

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CA2853379A1 (en) 2013-05-02
EP2797613A4 (en) 2015-09-09
KR20140092320A (ko) 2014-07-23
BR112014010091B1 (pt) 2022-11-08
WO2013063383A2 (en) 2013-05-02
CN104321069A (zh) 2015-01-28
US20160108099A1 (en) 2016-04-21
WO2013063383A3 (en) 2013-06-20
JP2015501156A (ja) 2015-01-15
ES2774779T3 (es) 2020-07-22
CA2853379C (en) 2020-11-24
RU2664673C2 (ru) 2018-08-21
US10040835B2 (en) 2018-08-07
EP2797613B1 (en) 2019-12-04
JP6293664B2 (ja) 2018-03-14
RU2014121256A (ru) 2015-12-10
MX354516B (es) 2018-03-08
KR102000141B1 (ko) 2019-07-15
US20140328821A1 (en) 2014-11-06
CN110777124A (zh) 2020-02-11
AU2012321102B2 (en) 2016-07-14
EP2797613A2 (en) 2014-11-05
AU2012321102C1 (en) 2016-11-10
BR112014010091A2 (pt) 2020-10-27
AU2012321102A1 (en) 2013-05-16
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US9265813B2 (en) 2016-02-23
HK1202049A1 (en) 2015-09-18

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