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公开(公告)号：US11298410B2
公开(公告)日：2022-04-12
申请号：US15578372
申请日：2016-06-01
Inventor： Kamel Khalili , Wenhui Hu , Yonggang Zhang
Abstract: Compositions for specifically cleaving target sequences in retroviruses include nucleic acids encoding a Clustered Regularly Interspace Short Palindromic Repeat (CRISPR) associated endonuclease and a guide RNA sequence complementary to one or more target nucleic acid sequences in a retrovirus genome.
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公开(公告)号：US20220088127A1
公开(公告)日：2022-03-24
申请号：US17422628
申请日：2020-01-15
Inventor： Jennifer Gordon , Kamel Khalili , T. Dianne Langford , Taha Mohseni Ahooyi
Abstract: Compositions in the prevention or treatment of neurodegenerative diseases or disorders associated with human immunodeficiency virus infection include agents which modulate the interactions of HIV Tat and Bcl-2 associated athanogene (BAG) molecules.
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公开(公告)号：US11236389B2
公开(公告)日：2022-02-01
申请号：US15115807
申请日：2015-01-30
Inventor： Arthur M. Feldman , Douglas G. Tilley , Weizhong Zhu , Kamel Khalili , Walter J. Koch
IPC: C12Q1/6883 , G01N33/68 , A61K48/00
Abstract: Compositions are directed to BCL2-associated athanogene 3 (BAG3) molecules and agents which modulate expression of BAG3 molecules. Pharmaceutical composition for administration to patients, for example, patients with heart failure, comprise one or more BAG3 molecules or agents which modulate expression of BAG3. Methods of treatment and identifying candidate therapeutic agents are also provided.
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公开(公告)号：US20210405031A1
公开(公告)日：2021-12-30
申请号：US16467813
申请日：2017-12-07
Inventor： Kamel Khalili , Jennifer Gordon
IPC: G01N33/50 , A01K67/027
Abstract: Animal models that are permissive for human polyomaviruses and their uses for the screening of candidate agents are described.
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公开(公告)号：US10279014B2
公开(公告)日：2019-05-07
申请号：US15864309
申请日：2018-01-08
Applicant: Excision BioTherapeutics, Inc. , Temple University of the Commonwealth System of Higher Education
Inventor： Kamel Khalili , Thomas Malcolm , Kenneth I. Kohn
Abstract: A method of eliminating the risk of JCV activation in a subject undergoing immunosuppressive therapy, by administering an effective amount of a gene editing composition directed toward at least one target sequence in the JCV genome, cleaving the target sequence in the JCV genome, disrupting the JCV genome, eliminating the JCV infection, eliminating the risk of JCV activation, and treating the subject with an immunosuppressive therapy. A pharmaceutical composition including at least one isolated nucleic acid sequence encoding a CRISPR-associated endonuclease and at least one gRNA having a spacer sequence complementary to a target sequence in a JCV DNA, the isolated nucleic acid sequences being included in at least one expression vector. Pharmaceutical compositions including at least one isolated nucleic acid sequence encoding at least one TALEN, at least one ZFN, and gene editing composition of C2c1, C2c3, TevCas9, Archaea Cas9, CasY.1-CasY.6, CasX, or argonaute protein, which target at least one nucleotide sequence of the JCV genome.
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Patent Agency Ranking
公开(公告)号：US20190093091A1
公开(公告)日：2019-03-28
申请号：US16091874
申请日：2017-03-29
Inventor： Kamel Khalili , Hassen Wollebo
Abstract: Compositions that specifically cleave target sequences in Flavivirus, for example Zika virus, include nucleic acids encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) associated endonuclease and a guide RNA sequence complementary to a target sequence in a Zika virus. These compositions are administered to a subject for treating an infection or at risk for contracting a Zika virus infection.
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公开(公告)号：US20190085326A1
公开(公告)日：2019-03-21
申请号：US16208314
申请日：2018-12-03
Inventor： Kamel Khalili , Wenhui Hu , Rafal Kaminski , Thomas Malcolm
Abstract: A CRISPR-endonuclease gene editing composition includes a guide RNA (gRNA) for targeting a specific viral sequence for cleavage by the endonuclease which introduces breaks in the double stranded DNA identified by the gRNA. Placing the gene encoding Cas9 under the control of a minimal promoter of, for example, HIV spanning the 5′-LTR, results in the activation by the HIV-1 transactivator protein, Tat. Co-expression of both a multiplex of, for example, HIV-specific gRNAs and endonuclease, e.g. Cas9, in cells results in the modification and/or excision of the segment of viral DNA, leading to the eradication of the virus in vitro and in vivo.
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公开(公告)号：US20180303915A1
公开(公告)日：2018-10-25
申请号：US15918107
申请日：2018-03-12
Inventor： Kamel Khalili , Wenhui HU
CPC classification number: A61K38/465 , A61K9/0034 , A61K35/12 , A61K45/06 , A61K48/00 , A61K48/005 , C12N7/00 , C12N9/22 , C12N15/111 , C12N2310/20 , C12N2320/30 , C12N2740/16063 , C12Y301/21
Abstract: A method of treating a subject at risk for having an HIV-1 virus infection, by administering to the subject a prophylactically effective amount of a composition comprising a CRISPR-associated endonuclease, and two or more different multiplex guide RNAs (gRNAs), wherein each of the at least two gRNAs is complementary to a different target nucleic acid sequence in a long terminal repeat (LTR) of proviral DNA of the virus that is unique from the genome of the host cell, cleaving a double strand of the proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease, cleaving a double strand of the proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease, excising an entire HIV-1 proviral genome, and eradicating the HIV-1 proviral DNA from the host cell and preventing HIV-1 retroviral infection.
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公开(公告)号：US12122997B2
公开(公告)日：2024-10-22
申请号：US15998558
申请日：2017-02-13
Inventor： Kamel Khalili , Wenhui Hu
IPC: C12N15/11 , A61K48/00 , C12N9/22 , C12N15/113
CPC classification number: C12N15/11 , A61K48/00 , C12N9/22 , C12N15/1132 , A01K2227/105 , A01K2267/0337 , C07K2319/09 , C12N2310/20 , C12N2750/14143
Abstract: Compositions for the in vivo delivery of a gene editing CRISPR/Cas9 complex was developed to eliminate integrated retroviral DNA sequences from latently infected human cells and animal disease models.
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公开(公告)号：US20230233654A1
公开(公告)日：2023-07-27
申请号：US17932528
申请日：2022-09-15
Applicant: Excision BioTherapeutics, Inc. , Temple University of the Commonwealth System of Higher Education
Inventor： Kamel Khalili , Thomas Malcolm , Kenneth I. Kohn
IPC: A61K38/46 , C12Q1/70 , C12N15/10 , C12N15/00 , A61P31/20 , A61K39/395 , C07K16/28 , C12N15/11 , A61K48/00
CPC classification number: A61K38/465 , C12Q1/70 , C12N15/102 , C12N15/00 , A61P31/20 , A61K39/39541 , C07K16/2839 , C12N15/11 , A61K48/00 , Y02A50/30 , C12N2740/15041 , C12N2799/00 , C12N2310/20 , A61K2039/505
Abstract: A method of eliminating the risk of JCV activation in a subject undergoing immunosuppressive therapy, by administering an effective amount of a gene editing composition directed toward at least one target sequence in the JCV genome, cleaving the target sequence in the JCV genome, disrupting the JCV genome, eliminating the JCV infection, eliminating the risk of JCV activation, and treating the subject with an immunosuppressive therapy. A pharmaceutical composition including at least one isolated nucleic acid sequence encoding a CRISPR-associated endonuclease and at least one gRNA having a spacer sequence complementary to a target sequence in a JCV DNA, the isolated nucleic acid sequences being included in at least one expression vector. Pharmaceutical compositions including at least one isolated nucleic acid sequence encoding at least one TALEN, at least one ZFN, and gene editing composition of C2c1, C2c3, TevCas9, Archaea Cas9, CasY.1-CasY.6, CasX, or argonaute protein, which target at least one nucleotide sequence of the JCV genome.
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