公开(公告)号：US20230138766A1

公开(公告)日：2023-05-04

申请号：US17915548

申请日：2021-03-30

Applicant: University of Massachusetts ,  Sichuan University

Inventor： Guangping Gao ,  Guangchao Xu ,  Phillip Tai ,  Yuquan Wei ,  Li Luo

IPC: C12N15/86 ,  A61K9/00 ,  C07K14/005

Abstract: Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., a transgene encoding one or more gene products) to a target cell. The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropisms for certain cell types (e.g., neurons, muscle cells, bone cells, heart cells, etc.). In some embodiments, recombinant A A Vs (rAAVs) comprising the capsid protein variants (e.g., AAVv66, SEQ ID NO: 1) are more efficiently packaged than rAAVs having certain wild-type AAV capsid proteins. Methods of delivering an rAAV comprising the AAV capsid protein variants are also described by the disclosure.

公开(公告)号：US20200316221A1

公开(公告)日：2020-10-08

申请号：US16842679

申请日：2020-04-07

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Guangchao Xu ,  Phillip Tai ,  Yuquan Wei ,  Li Luo

IPC: A61K48/00 ,  A61K9/00 ,  A61K35/76

Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

公开(公告)号：US20240414062A9

公开(公告)日：2024-12-12

申请号：US18432467

申请日：2024-02-05

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip Tai ,  Elisabet C. Mandon ,  Jianzhong Ai

IPC: H04L41/12 ,  H04L41/22

Abstract: In some aspects, the disclosure provides recombinant AAV and nucleic acid constructs having novel inverted terminal repeats (ITRs), cap, and/or rep genes. In some aspects, the disclosure relates to gene transfer methods using rAAVs described herein.

公开(公告)号：US20230048017A1

公开(公告)日：2023-02-16

申请号：US17685457

申请日：2022-03-03

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip Tai ,  Claudio Punzo ,  Haijiang Lin

IPC: C12N15/86 ,  C07K14/005 ,  C12N7/00 ,  A61K35/76 ,  A61P9/00

Abstract: Aspects of the disclosure relate to compositions and methods for expressing anti-Vascular endothelial cell growth factor (VEGF) agent in a cell or subject. In some embodiments, the disclosure provides rAAVs comprising a capsid protein (e.g., AAV2 variants, AAV2/3 hybrid variants, AAV8 variants, etc.), and a transgene encoding an anti-VEGF agent (e.g., KH902) and one or more regulatory sequences. In some embodiments, compositions described herein are useful for treating subjects having diseases associated with angiogenesis or aberrant VEGF activity/signaling.

公开(公告)号：US11578340B2

公开(公告)日：2023-02-14

申请号：US16341504

申请日：2017-10-13

Applicant: University of Massachusetts ,  Sichuan University

Inventor： Guangping Gao ,  Guangchao Xu ,  Phillip Tai ,  Yuquan Wei ,  Li Luo

IPC: C12N15/86 ,  A01K67/027 ,  A61K35/761 ,  C07K14/005 ,  C12N15/861 ,  C07K14/015 ,  A61K48/00

Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

公开(公告)号：US20210380981A1

公开(公告)日：2021-12-09

申请号：US17342587

申请日：2021-06-09

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Yi Lu ,  Qiang Zheng ,  Xun Xu ,  Phillip D. Zamore ,  Phillip Tai

IPC: C12N15/113 ,  C12N15/86 ,  C12N15/11 ,  A61P27/02 ,  A61K9/00

Abstract: The disclosure relates, in some aspects, to compositions and methods for treating corneal disease (e.g., corneal neovascularization. In some embodiments, the disclosure relates to rAAV-mediated delivery of an cornea-associated transgene to a subject. In some embodiments, the rAAV transduces the corneal tissue of a subject.

公开(公告)号：US20240243975A1

公开(公告)日：2024-07-18

申请号：US18432467

申请日：2024-02-05

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip Tai ,  Elisabet C. Mandon ,  Jianzhong Ai

IPC: H04L41/12 ,  H04L41/22

CPC classification number: H04L41/12 ,  H04L41/22

Abstract: In some aspects, the disclosure provides recombinant AAV and nucleic acid constructs having novel inverted terminal repeats (ITRs), cap, and/or rep genes. In some aspects, the disclosure relates to gene transfer methods using rAAVs described herein.

公开(公告)号：US20240207441A1

公开(公告)日：2024-06-27

申请号：US18288216

申请日：2022-04-26

Applicant: University of Massachusetts

Inventor： Yukiko Maeda ,  Phillip Tai ,  Guangping Gao

IPC: A61K48/00 ,  C07K14/005 ,  C12N15/86

CPC classification number: A61K48/0041 ,  A61K48/0075 ,  C07K14/005 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

Abstract: Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., an anti-inflammatory transgene encoding one or more gene products) to a joint. The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropisms for joint tissues (e.g., cartilage, skin, muscle, and bone cells). Methods of treating arthritis in a joint by administering an rAAV comprising the AAV capsid protein variants are also described by the disclosure.

公开(公告)号：US11851657B2

公开(公告)日：2023-12-26

申请号：US17342587

申请日：2021-06-09

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Yi Lu ,  Qiang Zheng ,  Xun Xu ,  Phillip D. Zamore ,  Phillip Tai

IPC: C07H21/04 ,  C12N15/113 ,  C12N15/86 ,  C12N15/11 ,  A61P27/02 ,  A61K9/00 ,  A61K48/00

CPC classification number: C12N15/113 ,  A61K9/0019 ,  A61K9/0048 ,  A61P27/02 ,  C12N15/111 ,  C12N15/86 ,  A61K48/00 ,  C12N2310/113 ,  C12N2310/141 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2750/14122 ,  C12N2750/14143

Abstract: The disclosure relates, in some aspects, to compositions and methods for treating corneal disease (e.g., corneal neovascularization. In some embodiments, the disclosure relates to rAAV-mediated delivery of an cornea-associated transgene to a subject. In some embodiments, the rAAV transduces the corneal tissue of a subject.

公开(公告)号：US20230374545A1

公开(公告)日：2023-11-23

申请号：US18150477

申请日：2023-01-05

Applicant: University of Massachusetts ,  Sichuan University

Inventor： Guangping Gao ,  Guangchao Xu ,  Phillip Tai ,  Yuquan Wei ,  Li Luo

IPC: C12N15/86 ,  C12N15/861 ,  C07K14/005 ,  C07K14/015 ,  A61K35/761 ,  A01K67/027 ,  A61K48/00

CPC classification number: C12N15/86 ,  C12N15/861 ,  C07K14/005 ,  C07K14/015 ,  A61K35/761 ,  A01K67/0275 ,  C12N2750/14122 ,  C12N2750/14123 ,  A01K2267/03 ,  A01K2217/05 ,  A61K48/00 ,  C12N2750/14143 ,  C12N2750/14145

Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.