公开(公告)号：US20180208914A1

公开(公告)日：2018-07-26

申请号：US15873483

申请日：2018-01-17

Applicant: EXCISION BIOTHERAPEUTICS, INC.

Inventor： Thomas Malcolm ,  Kamel Khalili

IPC: C12N9/22 ,  C12N7/00 ,  A61P31/14 ,  A61P31/22 ,  A61P31/18 ,  A61P31/20 ,  C12N15/11 ,  C12N9/96 ,  C12N15/113 ,  A61K38/46 ,  A61K31/713

CPC classification number: C12N9/22 ,  A61K31/713 ,  A61K38/465 ,  A61K48/00 ,  A61P31/14 ,  A61P31/18 ,  A61P31/20 ,  A61P31/22 ,  C12N7/00 ,  C12N9/96 ,  C12N15/11 ,  C12N15/1131 ,  C12N15/1132 ,  C12N15/1133 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2320/31 ,  C12N2740/15043 ,  C12N2740/16032 ,  C12N2740/16043 ,  C12N2800/80 ,  Y02A50/385 ,  Y02A50/387 ,  Y02A50/389 ,  Y02A50/391 ,  Y02A50/463 ,  Y02A50/467

Abstract: A composition for treating a lysogenic virus, including a lentiviral vector encoding isolated nucleic acid encoding two or more gene editors chosen from gene editors that target viral DNA, gene editors that target viral RNA, and combinations thereof. A composition for treating a lytic virus, including a lentiviral vector encoding isolated nucleic acid encoding at least one gene editor that targets viral DNA and a viral RNA targeting composition. A composition for treating both lysogenic and lytic viruses, including a lentiviral vector encoding isolated nucleic acid encoding two or more gene editors that target viral RNA. A composition for treating lytic viruses. Methods of treating a lysogenic virus or a lytic virus, by administering the above compositions to an individual having a virus and inactivating the virus.

公开(公告)号：US20190194652A1

公开(公告)日：2019-06-27

申请号：US16290343

申请日：2019-03-01

Applicant: EXCISION BIOTHERAPEUTICS. INC.

Inventor： Thomas MALCOLM

IPC: C12N15/11 ,  C12N9/22 ,  A61K9/00 ,  C12Q1/70 ,  G01N33/569

CPC classification number: C12N9/22 ,  C12N15/102

Abstract: A method of performing a clinical trial for a gene editing or gene excising system for treating HIV in humans, by recruiting HIV infected individuals currently receiving highly active antiretroviral therapy (HAART) that is effective in lowering viral load and entering qualified individuals as participants in a clinical trial, administering the gene editing or gene excising system treatment to the participants in Phase 1a, Phase 1b, and Phase 1c, and performing assays to confirm HIV viral genome excision from the participants' cells. A method of performing a clinical trial for a gene editing or gene excising system for treating a latent viral infection in humans.

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

IPC: C12Q1/70 ,  A61K38/46 ,  A61K39/00 ,  A61K48/00 ,  A61P31/20 ,  C07K16/28 ,  C12N15/11 ,  A61K39/395

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.

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.

公开(公告)号：US20180228921A1

公开(公告)日：2018-08-16

申请号：US15895414

申请日：2018-02-13

Applicant: EXCISION BIOTHERAPEUTICS, INC.

Inventor： Kamel Khalili ,  Won-Bin Young

IPC: A61K49/00 ,  A61K38/46 ,  A61K31/7088 ,  A61P31/18

CPC classification number: A61K49/0013 ,  A01K2227/105 ,  A01K2267/0337 ,  A01K2267/0393 ,  A61K31/7088 ,  A61K38/465 ,  A61K48/00 ,  A61K49/0097 ,  A61P31/18 ,  C12N2740/16043 ,  Y02A50/385 ,  Y02A50/387 ,  Y02A50/389 ,  Y02A50/391 ,  Y02A50/393 ,  Y02A50/397 ,  Y02A50/463 ,  Y02A50/467

Abstract: A reporter gene that can be administered to an individual such as HIV-BAL-eLuc in order to detect the presence of a virus. A method of detecting the presence of virus in an individual, by administering a reporter gene to the individual, associating the reporter gene with the virus, imaging the individual, and detecting the presence of virus in the individual. A method of determining the efficacy of a treatment for a virus, by administering a reporter gene to the individual receiving treatment for the virus, associating the reporter gene with the virus, imaging the individual, detecting the presence of virus in the individual, and determining if the treatment is effective.

公开(公告)号：US20190285632A1

公开(公告)日：2019-09-19

申请号：US16300654

申请日：2017-05-24

Applicant: Excision Biotherapeutics, Inc.

Inventor： Thomas Malcolm

IPC: G01N33/569

Abstract: A diagnostic panel including a test for detecting at least one biomarker that indicates the presence of a virus. A kit including the diagnostic panel, instructions for use, materials to take and apply samples to the panel, and descriptions of biomarker levels and their meaning. A method of detecting the presence of disease, by taking a sample of an individual, applying the sample to the diagnostic panel including at least one biomarker indicative of disease, detecting the presence of at least one biomarker, comparing levels of the biomarker to a baseline, and determining if the individual has a disease. Methods of determining the stage of a disease, monitoring the progress of disease treatments, determining viral suppression or rebound, and detecting latent virus.

公开(公告)号：US20190038770A1

公开(公告)日：2019-02-07

申请号：US16072636

申请日：2017-01-24

Applicant: EXCISION BIOTHERAPEUTICS,INC. ,  Temple University of the Commonwealth System of Higher Education

Inventor： Kamel KHALILI ,  Thomas MALCOLM

IPC: A61K48/00 ,  C12N15/66 ,  C12N15/113 ,  C12N15/861 ,  A61P31/20

Abstract: The present invention includes methods and compositions for elimination of polyoma viruses, such as John Cunningham Virus (JVC), from host cells, and the treatment of polyoma-virus related diseases, such as progressive multifocal leukoencephalopathy (PML). The compositions include isolated nucleic acid sequences comprising a CRISPR-associated endonuclease and a guide RNA, wherein the guide RNA is complementary to a target sequence in a polyoma virus.

公开(公告)号：US20190032057A1

公开(公告)日：2019-01-31

申请号：US16072589

申请日：2017-01-24

Applicant: EXCISION BIOTHERAPEUTICS, INC. ,  Temple University of the Commonwealth System of Higher Education

Inventor： Kamel KHALILI ,  Thomas Malcolm

IPC: C12N15/113 ,  C12N15/10 ,  C12Q1/70 ,  A61P31/18 ,  C12N15/86 ,  C12N9/22 ,  A61K31/711 ,  C12N15/70

Abstract: A method of inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus by treating the host cell with a composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, and two or more different 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) in the proviral DNA, and inactivating the proviral DNA. A composition for use in inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus including isolated nucleic acid sequences comprising a CRISPR-associated endonuclease and a guide RNA, wherein the guide RNA is complementary to a target sequence in a human immunodeficiency virus.

公开(公告)号：US20180140682A1

公开(公告)日：2018-05-24

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

IPC: A61K38/46 ,  C12N15/11 ,  A61K39/395 ,  C07K16/28 ,  A61P31/20

CPC classification number: A61K38/465 ,  A61K39/39541 ,  A61K2039/505 ,  A61P31/20 ,  C07K16/2839 ,  C12N15/102 ,  C12N15/11 ,  C12N2310/20 ,  C12Q1/70 ,  Y02A50/467

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.

公开(公告)号：US20230048681A1

公开(公告)日：2023-02-16

申请号：US17744475

申请日：2022-05-13

Applicant: Excision BioTherapeutics Inc

Inventor： Thomas MALCOLM

IPC: C12N15/113 ,  C12N9/22

Abstract: A composition for treating a lysogenic virus, including isolated nucleic acid encoding two or more gene editors chosen from gene editors that target viral DNA, gene editors that target viral RNA, and combinations thereof. A composition for treating a lytic virus, including isolated nucleic acid encoding at least one gene editor that targets viral DNA and a viral RNA targeting composition. A composition for treating both lysogenic and lytic viruses, including isolated nucleic acid encoding two or more gene editors that target viral RNA, chosen from CRISPR-associated nucleases, Argonaute endonuclease gDNAs, C2c2, RNase P RNA, and combinations thereof. A composition for treating lytic viruses, including isolated nucleic acid encoding two or more gene editors that target viral RNA and a viral RNA targeting composition. Methods of treating a lysogenic virus or a lytic virus, by administering the above compositions to an individual having a virus and inactivating the virus.