公开(公告)号：US11884926B2

公开(公告)日：2024-01-30

申请号：US16966504

申请日：2019-02-01

Applicant: University of Massachusetts

Inventor： Terence Flotte ,  Qiushi Tang ,  Allison Keeler-Klunk ,  Qin Su

IPC: C12N15/70 ,  C12N7/00

CPC classification number: C12N15/70 ,  C12N7/00 ,  C12N2750/14143 ,  C12N2750/14151 ,  C12N2750/14171

Abstract: In some aspects, the disclosure relates to compositions and methods for production of recombinant adeno-associated virus (rAAV) particles. The disclosure is based, in part, on isolated nucleic acids and systems that include only two vectors and are suitable for production of multiple different single gene viral vectors in a multiple small-scale campaign mode (e.g., 1013 to 1016 viral particles.)

公开(公告)号：US20210393805A1

公开(公告)日：2021-12-23

申请号：US17054812

申请日：2019-05-16

Applicant: University of Massachusetts

Inventor： Alisha Gruntman ,  Terence Flotte

IPC: A61K48/00 ,  C12N15/86

Abstract: In some aspects, the disclosure relates to methods and compositions for delivering a transgene to a subject. The disclosure is based, in part, on compositions (e.g., viral vectors, such as rAAV vectors) and methods of venous limb perfusion (VLP) that efficiently transduce muscle tissue and enhance serum concentrations of secreted transgenes.

公开(公告)号：US20190211327A1

公开(公告)日：2019-07-11

申请号：US16059121

申请日：2018-08-09

Applicant: University of Massachusetts

Inventor： Terence Flotte ,  Christian Mueller ,  Phillip D. Zamore

IPC: C12N15/113 ,  A61K48/00 ,  A61K38/57 ,  A61K31/713 ,  C07K14/81 ,  A61K35/12 ,  C12N7/00 ,  C12N15/86 ,  C12N15/67

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K35/12 ,  A61K38/57 ,  A61K48/005 ,  A61K48/0058 ,  C07K14/8125 ,  C12N7/00 ,  C12N15/111 ,  C12N15/67 ,  C12N15/86 ,  C12N2310/141 ,  C12N2310/3519 ,  C12N2320/31 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143

Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

公开(公告)号：US10035825B2

公开(公告)日：2018-07-31

申请号：US15007559

申请日：2016-01-27

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Terence Flotte ,  Jun Xie

IPC: C12N7/00 ,  A61K39/23 ,  C07K14/005 ,  C12N15/86 ,  A61K31/7088 ,  A61K35/76 ,  A61K48/00

CPC classification number: C07K14/005 ,  A61K31/7088 ,  A61K35/76 ,  A61K48/00 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

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

公开(公告)号：US20240240180A1

公开(公告)日：2024-07-18

申请号：US18426444

申请日：2024-01-30

Applicant: University of Massachusetts

Inventor： Terence Flotte ,  Christian Mueller ,  Phillip D. Zamore

IPC: C12N15/113 ,  A61K31/713 ,  A61K35/12 ,  A61K38/57 ,  A61K48/00 ,  C07K14/81 ,  C12N7/00 ,  C12N15/67 ,  C12N15/86 ,  C12N15/11

CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K35/12 ,  A61K38/57 ,  A61K48/005 ,  A61K48/0058 ,  C07K14/8125 ,  C12N7/00 ,  C12N15/67 ,  C12N15/86 ,  C12N15/111 ,  C12N2310/141 ,  C12N2310/3519 ,  C12N2320/31 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143

Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

公开(公告)号：US11254939B2

公开(公告)日：2022-02-22

申请号：US16795757

申请日：2020-02-20

Applicant: University of Massachusetts

Inventor： Terence Flotte ,  Christian Mueller ,  Phillip D. Zamore

IPC: C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  C12N15/67 ,  A61K48/00 ,  A61K35/12 ,  C12N7/00 ,  C12N15/86 ,  A61K31/713 ,  A61K38/57 ,  C07K14/81 ,  C12N15/11

Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

公开(公告)号：US20200024617A1

公开(公告)日：2020-01-23

申请号：US16584616

申请日：2019-09-26

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Jun Xie ,  Terence Flotte

IPC: C12N15/86 ,  C07K14/005 ,  A01K67/027 ,  A61K48/00 ,  C12N7/00 ,  C12N15/113 ,  C12N15/85

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：US20170369874A9

公开(公告)日：2017-12-28

申请号：US15098833

申请日：2016-04-14

Applicant: University of Massachusetts

Inventor： Terence Flotte ,  Christian Mueller

IPC: C12N15/113 ,  A61K38/57 ,  A61K48/00 ,  C12N7/00 ,  A61K31/713 ,  C12N15/67 ,  C07K14/81 ,  A61K35/12 ,  C12N15/86 ,  C12N15/11

CPC classification number: C12N15/86 ,  A61K31/713 ,  A61K35/12 ,  A61K38/57 ,  A61K48/005 ,  A61K48/0058 ,  C07K14/8125 ,  C12N7/00 ,  C12N15/111 ,  C12N15/113 ,  C12N15/67 ,  C12N2310/141 ,  C12N2310/3519 ,  C12N2320/31 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143

Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

公开(公告)号：US20160222067A1

公开(公告)日：2016-08-04

申请号：US15007559

申请日：2016-01-27

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Terence Flotte ,  Jun Xie

IPC: C07K14/005 ,  A61K35/76 ,  A61K31/7088 ,  C12N7/00

CPC classification number: C07K14/005 ,  A61K31/7088 ,  A61K35/76 ,  A61K48/00 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

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

Abstract translation: 本发明在某些方面涉及具有不同组织靶向能力的重组腺相关病毒。 在一些方面，本发明涉及使用重组腺相关病毒的基因转移方法。 在一些方面，本发明涉及分离的AAV衣壳蛋白和编码其的分离的核酸。

公开(公告)号：US20210047650A1

公开(公告)日：2021-02-18

申请号：US16966504

申请日：2019-02-01

Applicant: University of Massachusetts

Inventor： Terence Flotte ,  Qiushi Tang ,  Allison Keeler-Klunk ,  Qin Su

IPC: C12N15/70 ,  C12N7/00

Abstract: In some aspects, the disclosure relates to compositions and methods for production of recombinant adeno-associated virus (rAAV) particles. The disclosure is based, in part, on isolated nucleic acids and systems that include only two vectors and are suitable for production of multiple different single gene viral vectors in a multiple small-scale campaign mode (e.g., 1013 to 1016 viral particles.)