公开(公告)号：US11285193B2

公开(公告)日：2022-03-29

申请号：US15882207

申请日：2018-01-29

Applicant: Temple University of the Commonwealth System of Higher Education

Inventor： Kamel Khalili ,  Wenhui Hu

IPC: A61K38/46 ,  C12N15/11 ,  A61K48/00 ,  A61K9/00 ,  A61K35/12 ,  A61K45/06 ,  C12N7/00 ,  C12N9/22

Abstract: A method of treating a subject at risk for having a virus infection, by administering to the subject a prophylactically effective amount of a composition comprising a vector encoding a CRISPR-associated endonuclease and at least two guide RNAs, wherein the guide RNAs are complementary to two target sequences spanning from the 5′- to 3′-LTRs of the sequence in the virus, and preventing a retroviral infection.

公开(公告)号：US11273209B2

公开(公告)日：2022-03-15

申请号：US16875271

申请日：2020-05-15

Applicant: Temple University—of the Commonwealth System of Higher Education

Inventor： Kamel Khalili ,  Wenhui Hu ,  Yonggang Zhang

IPC: A61K38/46 ,  C12N15/113 ,  A61K48/00 ,  A61P31/18 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90

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.

公开(公告)号：US20210060138A1

公开(公告)日：2021-03-04

申请号：US16812140

申请日：2020-03-06

Applicant: Temple University - of the Commonwealth System of Higher Education ,  BOARD OF REGENTS OF THE UNIVERSITY OF NEBRASKA

Inventor： Kamel Khalili ,  Howard E. Gendelman ,  Benson Edagwa

IPC: A61K38/46 ,  A61K35/761 ,  A61P31/18 ,  A61K31/5365 ,  A61K31/7068 ,  A61K31/7076 ,  A61K31/505 ,  A61K31/7105

Abstract: Methods of eliminating a retrovirus from a subject utilize nanoformulated anti-retroviral compounds and gene editing agents. Compositions comprise at least one anti-retroviral compounds, at least one gene-editing agent, or combinations thereof.

公开(公告)号：US20190183971A1

公开(公告)日：2019-06-20

申请号：US16324719

申请日：2017-08-10

Applicant: Temple University - of the Commonwealth System of Higher Education

Inventor： Arthur M. Feldman ,  Joseph Y. Cheung ,  Kamel Khalili

IPC: A61K38/17 ,  A61P9/10 ,  C12N15/85 ,  A61K31/69 ,  A61K9/00

CPC classification number: A61K38/1709 ,  A61K9/0019 ,  A61K31/69 ,  A61K38/05 ,  A61K45/06 ,  A61P9/10 ,  C12N15/85 ,  A61K2300/00

Abstract: Compositions for treatment and prevention of ischemia/reperfusion injury include agents that increase levels of the Bcl2-associated athanogene 3 (BAG3). The compositions are administered to a subject suffering from ischemia/reperfusion injury or who is at risk for ischemia/reperfusion injury.

公开(公告)号：US20190093092A1

公开(公告)日：2019-03-28

申请号：US16099098

申请日：2016-12-12

Applicant: Temple University - of the Commonwealth System of Higher Education

Inventor： Kamel Khalili ,  Hassen Wollebo

IPC: C12N9/22 ,  C12N15/11 ,  A61K38/46

Abstract: Compositions that specifically cleave target sequences in Herpesviridae, for example Varicella zoster virus (VZV) 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 VZV. These compositions are administered to a subject for treating an infection or at risk for contracting a VZV infection.

公开(公告)号：US20180221458A1

公开(公告)日：2018-08-09

申请号：US15942773

申请日：2018-04-02

Applicant: Temple University of the Commonwealth System of Higher Education

Inventor： Kamel Khalili ,  Wenhui Hu

IPC: A61K38/46 ,  C12N15/11 ,  A61K35/12 ,  A61K48/00 ,  C12N7/00 ,  C12N9/22 ,  A61K9/00 ,  A61K45/06

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 having or at risk for having an HIV-1 virus infection, by administering to the subject a therapeutically 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, eradicating the HIV-1 proviral DNA from the host cell, and completely resolving the symptoms of HIV-1, decreasing the severity of the symptoms of HIV-1, or slowing HIV-1's progression.

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：US12251429B2

公开(公告)日：2025-03-18

申请号：US17017037

申请日：2020-09-10

Applicant: Temple University—of the Commonwealth System of Higher Education

Inventor： Kamel Khalili ,  Wenhui Hu ,  Yonggang Zhang

IPC: A61K38/46 ,  A61K48/00 ,  A61P31/18 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  C12N15/90

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.

公开(公告)号：US20250082778A1

公开(公告)日：2025-03-13

申请号：US18921813

申请日：2024-10-21

Applicant: Temple University - of the Commonwealth System of Higher Education

Inventor： Kamel Khalili ,  Wenhui Hu ,  Hassen Wollebo

IPC: A61K48/00 ,  C12N9/22 ,  C12N15/113 ,  C12N15/63

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

公开(公告)号：US20230390367A1

公开(公告)日：2023-12-07

申请号：US18249569

申请日：2021-10-19

Applicant: Temple University - of the Commonwealth System of Higher Education

Inventor： Kamel Khalili ,  Rafal Kaminski ,  Shuren Liao

IPC: A61K38/46 ,  C12N15/113 ,  A61K45/06

CPC classification number: A61K38/465 ,  C12N15/1131 ,  A61K45/06 ,  C12N2310/20

Abstract: Compositions include gene editing agents, e.g. CRISPR that employ Cas13a for editing and inactivating sequences in the Coronavirus genome.