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公开(公告)号：US20210371481A1
公开(公告)日：2021-12-02
申请号：US17204627
申请日：2021-03-17
摘要： The present invention provides methods and compositions for treating and/or preventing age related macular degeneration and other conditions involving macular degeneration, ocular neovascularization, or ocular inflammation. In an exemplary embodiment, a method is disclosed that involves administering an expression vector that delivers a secretable and cell penetrating CARD to a subject in need of treatment or prevention of age-related macular degeneration or another condition involving macular degeneration or ocular neovascularization.
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公开(公告)号：US20210324387A1
公开(公告)日：2021-10-21
申请号：US17327609
申请日：2021-05-21
申请人： University of Florida Research Foundation, Incorporated , The Trustees of the University of Pennsylvania
发明人： Alfred S. Lewin , William W. Hauswirth , Michael T. Massengill , William Beltran , Gustavo D. Aguirre , Artur Cideciyan , Samuel Jacobson
IPC分类号： C12N15/113 , C12N15/86 , A61P27/02 , C12N7/00
摘要： Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.
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公开(公告)号：US10981961B2
公开(公告)日：2021-04-20
申请号：US14775289
申请日：2014-03-11
摘要： The present invention provides methods and compositions for treating and/or preventing age related macular degeneration and other conditions involving macular degeneration, ocular neovascularization, or ocular inflammation. In an exemplary embodiment, a method is disclosed that involves administering an expression vector that delivers a secretable and cell penetrating CARD to a subject in need of treatment or prevention of age-related macular degeneration or another condition involving macular degeneration or ocular neovascularization.
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公开(公告)号：US20190093111A1
公开(公告)日：2019-03-28
申请号：US16081307
申请日：2017-03-01
申请人： University of Florida Research Foundation, Incorporated , The Trustees of the University of Pennsylvania
发明人： Alfred S. Lewin , William W. Hauswirth , MIchael T. Massengill , William Beltran , Gustavo D. Aguirre , Artur Cideciyan , Samuel Jacovson
IPC分类号： C12N15/113 , C12N7/00 , A61P27/02
摘要： Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.
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公开(公告)号：US20210207147A1
公开(公告)日：2021-07-08
申请号：US17059815
申请日：2019-06-03
申请人： University of Florida Research Foundation, Incorporated , The Trustees of the University of Pennsylvania
发明人： Alfred S. Lewin , William W. Hauswirth , Michael T. Massengill , William Beltran , Gustavo D. Aguirre , Artur Cideciyan , Samuel Jacobson
IPC分类号： C12N15/113 , C12N15/86
摘要： Aspects of the disclosure relate to methods and compositions useful for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering RNA to a subject in order to reduce expression of one or both alleles of an endogenous RHO gene (for example a mutant rho allele associated with retinitis pigmentosa) in a subject. In some embodiments, a replacement RHO coding sequence that is resistant to the interfering RNA also is delivered to the subject.
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公开(公告)号：US20210000973A1
公开(公告)日：2021-01-07
申请号：US16978800
申请日：2019-03-08
摘要： In some aspects, the disclosure relates to compositions and methods that are useful for treating inflammatory disorders and conditions. In some embodiments, the disclosure provides recombinant adeno-associated virus (rAAV) vectors and particles encoding a transgene that expresses a Myxomavirus serine proteinase inhibitor (Serp) protein (e.g., Serp-1) and methods for treating inflammatory disorders using the same.
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公开(公告)号：US20240101607A1
公开(公告)日：2024-03-28
申请号：US18314773
申请日：2023-05-09
发明人： Douglas Grant McFadden , Alfred S. Lewin , Alexandra Rose Lucas , Cristhian J. Ildefonso , Mohammed Masmudur Rahman
CPC分类号： C07K14/005 , A61K9/0048 , A61K38/162 , A61K48/005 , C12N7/00 , C12N15/86 , A61K48/00 , C12N2710/24022 , C12N2750/14143
摘要： Disclosed are methods and compositions for preventing, treating, and/or ameliorating one or more symptoms of inflammation in a mammal. In particular, viral vectors and medicaments containing them are disclosed, which are useful in the prophylaxis, therapy, or amelioration of symptoms of one or more inflammatory-mediated mammalian diseases, such as age-related macular degeneration (AMD), arthritis, Bechet's disease, Best macular dystrophy, corneal inflammation, diabetic retinopathy, drusen formation, dry AMD, dry eye, geographic atrophy, glaucomaocular neovascularization, Lupus erythematosus, macular degeneration, Mallatia Leventinese and Doyne honeycomb retinal dystrophy, nephritis, ocular hypertension, ocular inflammation, recurrent uveitis, Sorsby fundus dystrophy, vasculitis, vitreoretinopathy, wet AMD, or related disorders. In exemplary methods, administration of a pharmaceutical composition comprising a recombinant viral vector that delivers a secretable and cell-penetrating M013 protein or peptide to a subject in need thereof facilitated treatment of particular human disorders such as AMD, ocular neovascularization, uveitis, and related inflammatory ocular disease.
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公开(公告)号：US11685767B2
公开(公告)日：2023-06-27
申请号：US15261599
申请日：2016-09-09
发明人： Douglas Grant McFadden , Alfred S. Lewin , Alexandra Rose Lucas , Cristhian J. Ildefonso , Mohammed Masmudur Rahman
IPC分类号： A61K48/00 , C07H21/04 , C12N15/86 , C07K14/005 , A61K9/00 , A61K38/16 , C12N7/00 , A01K67/00
CPC分类号： C07K14/005 , A61K9/0048 , A61K38/162 , A61K48/005 , C12N7/00 , C12N15/86 , A61K48/00 , C12N2710/24022 , C12N2750/14143
摘要： Disclosed are methods and compositions for preventing, treating, and/or ameliorating one or more symptoms of inflammation in a mammal. In particular, viral vectors and medicaments containing them are disclosed, which are useful in the prophylaxis, therapy, or amelioration of symptoms of one or more inflammatory-mediated mammalian diseases, such as age-related macular degeneration (AMD), arthritis, Bechet's disease, Best macular dystrophy, corneal inflammation, diabetic retinopathy, drusen formation, dry AMD, dry eye, geographic atrophy, glaucomaocular neovascularization, Lupus erythematosus, macular degeneration, Mallatia Leventinese and Doyne honeycomb retinal dystrophy, nephritis, ocular hypertension, ocular inflammation, recurrent uveitis, Sorsby fundus dystrophy, vasculitis, vitreoretinopathy, wet AMD, or related disorders. In exemplary methods, administration of a pharmaceutical composition comprising a recombinant viral vector that delivers a secretable and cell-penetrating M013 protein or peptide to a subject in need thereof facilitated treatment of particular human disorders such as AMD, ocular neovascularization, uveitis, and related inflammatory ocular disease.
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公开(公告)号：US20190231902A1
公开(公告)日：2019-08-01
申请号：US16312277
申请日：2017-06-21
申请人： University of Florida Research Foundation, Incorporated , Board of Supervisors of Louisiana State University & Agricultural & Mechanical College
发明人： David C. Bloom , Alfred S. Lewin , Donna M. Neumann , Zachary L. Watson , Sonal Sanjeev Tuli , Gregory Scott Schultz
IPC分类号： A61K48/00 , C12N5/0793 , C12N15/86 , C12N15/113 , A61P31/22
CPC分类号： A61K48/0091 , A61P31/22 , C12N5/062 , C12N15/1133 , C12N15/86 , C12N2310/121 , C12N2310/20 , C12N2320/32 , C12N2510/00 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
摘要： Aspects of the application relate to methods and compositions for delivering therapeutic nucleic acids to neural cells or tissue in a subject. Additional aspects of the application relate to therapeutic nucleic acids, for example therapeutic ribozymes, that are useful for inhibiting viral reactivation in a subject.
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公开(公告)号：US12060395B2
公开(公告)日：2024-08-13
申请号：US17342120
申请日：2021-06-08
CPC分类号： C07K14/4702 , A61K9/0048 , A61K38/1709 , A61K48/0075 , C07K2319/00 , C07K2319/02 , C07K2319/10 , C07K2319/50
摘要： The present invention provides methods and compositions for treating and/or preventing age related macular degeneration and other conditions involving macular degeneration, ocular neovascularization, or inflammation, including ocular inflammation. In some embodiments, the methods comprise administering an expression vector that delivers a secretable and cell penetrating Nrf2 to a subject in need thereof.
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