PL2797613T3 - Wektory kodujące pręcikowy czynnik żywotności czopków - Google Patents

Wektory kodujące pręcikowy czynnik żywotności czopków

Info

Publication number
PL2797613T3
PL2797613T3 PL12843778T PL12843778T PL2797613T3 PL 2797613 T3 PL2797613 T3 PL 2797613T3 PL 12843778 T PL12843778 T PL 12843778T PL 12843778 T PL12843778 T PL 12843778T PL 2797613 T3 PL2797613 T3 PL 2797613T3
Authority
PL
Poland
Prior art keywords
vectors encoding
viability factor
cone viability
derived cone
encoding rod
Prior art date
Application number
PL12843778T
Other languages
English (en)
Polish (pl)
Inventor
Tianci Luo
Original Assignee
Wellstat Ophthalmics Corporation
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Wellstat Ophthalmics Corporation filed Critical Wellstat Ophthalmics Corporation
Publication of PL2797613T3 publication Critical patent/PL2797613T3/pl

Links

Classifications

    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • 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
    • 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
    • A61P25/28—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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • 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
    • 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
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
    • A61P27/08—Mydriatics or cycloplegics
    • 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
    • A61P27/10—Ophthalmic agents for accommodation disorders, e.g. myopia
    • 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
    • A61P27/12—Ophthalmic agents for cataracts
    • 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
    • A61P27/14—Decongestants or antiallergics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/861—Adenoviral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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
    • 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
    • A61K48/0066—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
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
PL12843778T 2011-10-27 2012-10-26 Wektory kodujące pręcikowy czynnik żywotności czopków PL2797613T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161552155P 2011-10-27 2011-10-27
EP12843778.7A EP2797613B1 (en) 2011-10-27 2012-10-26 Vectors encoding rod-derived cone viability factor
PCT/US2012/062106 WO2013063383A2 (en) 2011-10-27 2012-10-26 Vectors encoding rod-derived cone viability factor

Publications (1)

Publication Number Publication Date
PL2797613T3 true PL2797613T3 (pl) 2020-06-15

Family

ID=48168788

Family Applications (1)

Application Number Title Priority Date Filing Date
PL12843778T PL2797613T3 (pl) 2011-10-27 2012-10-26 Wektory kodujące pręcikowy czynnik żywotności czopków

Country Status (14)

Country Link
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

Also Published As

Publication number Publication date
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
DK2797613T3 (da) 2020-03-02
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
MX2014004949A (es) 2014-09-12
US9265813B2 (en) 2016-02-23
HK1202049A1 (en) 2015-09-18

Similar Documents

Publication Publication Date Title
PL2797613T3 (pl) Wektory kodujące pręcikowy czynnik żywotności czopków
GB2501836B (en) Video encoding
DK3505720T3 (en) Cementering under styret tryk
GB201108879D0 (en) Vector
PL2914359T3 (pl) Separator magnetyczny
EP2721506A4 (en) RECONCILIATION OF EFFECTIVE STATUS
GB201402766D0 (en) Spillproof container assemblies
GB201109414D0 (en) Diathermy -ionisation technique
GB2492645B (en) Cryostat
GB2501958B (en) Backpack
EP2662268A4 (en) CAP MOUNTING STRUCTURE
PL3219801T3 (pl) Konstrukt
ZA201400624B (en) Solids-handling equipment
ZA201308491B (en) Solids-handling equipment
PL3193332T3 (pl) Kwantyzacja wektorowa
PL2831757T3 (pl) Wektorowy kwantyzator
PL2584028T3 (pl) Cząstka
GB201114045D0 (en) Magnet
GB201317652D0 (en) Magnetic separator
SG11201402471SA (en) Photocatalyst-supporting structure
GB201109834D0 (en) Rucksack
GB201107369D0 (en) Case structure
GB201114452D0 (en) Paca
GB201114623D0 (en) Foc
GB201115328D0 (en) Foc