公开(公告)号：US20240216542A1

公开(公告)日：2024-07-04

申请号：US18557102

申请日：2022-04-18

Applicant: University of Massachusetts ,  The Clinic for Special Children, Inc.

Inventor： Huiya Yang ,  Guangping Gao ,  Kevin A. Strauss ,  Dan Wang

IPC: A61K48/00 ,  A61P3/00 ,  A61P25/00 ,  C12N9/10 ,  C12N15/86

CPC classification number: A61K48/0058 ,  A61K48/0075 ,  A61P3/00 ,  A61P25/00 ,  C12N9/1081 ,  C12N15/86 ,  C12Y204/99009 ,  C12N2750/14143 ,  C12N2830/008

Abstract: Aspects of the disclosure relate to methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof by administering an rAAV expressing one or more Ganglioside GM3 synthase (GM3S) isoforms via intracerebroventricular (ICV) administration.

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：US20240207370A1

公开(公告)日：2024-06-27

申请号：US18288211

申请日：2022-04-26

Applicant: University of Massachusetts ,  The Clinic for Special Children, Inc

Inventor： Guangping Gao ,  Dan Wang ,  Jiaming Wang ,  Kevin A. Strauss

IPC: A61K38/44 ,  A61K48/00 ,  A61P3/00 ,  C12N15/86

CPC classification number: A61K38/44 ,  A61K48/0033 ,  A61K48/0066 ,  A61P3/00 ,  C12N15/86 ,  C12Y102/04004 ,  C12N2750/14143

Abstract: In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA protein, which is the E1-alpha subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, and/or BCKDHB, which is the E1-beta subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex in a subject. In some embodiments, the disclosure provides methods of treating a subject having Maple Syrup Urine Disease (MSUD).

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.

公开(公告)号：US20230340529A1

公开(公告)日：2023-10-26

申请号：US18024359

申请日：2021-09-02

Applicant: University of Massachusetts

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

IPC: C12N15/86 ,  A61K35/761

CPC classification number: C12N15/86 ,  A61K35/761 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2750/14122

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：US11542525B2

公开(公告)日：2023-01-03

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 ,  A61K35/00

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：US11413356B2

公开(公告)日：2022-08-16

申请号：US16093813

申请日：2017-04-14

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Dominic Gessler

IPC: A61K48/00 ,  A61K38/50 ,  A61K35/761 ,  A61K35/30 ,  G01N33/574 ,  C12N9/80 ,  C12N9/10 ,  A61K38/45 ,  A61K45/06

Abstract: In some aspects, the disclosure relates to compositions and methods useful for the diagnosis and treatment of diseases associated with a metabolic imbalance in a subject (e.g., cancer). In some embodiments, the methods comprise administering to a subject an N-acetylaspartate (NAA)-depleting agent or an N-acetylaspartate (NAA)-increasing agent based upon the subject's metabolic profile.

公开(公告)号：US20220162571A1

公开(公告)日：2022-05-26

申请号：US17602348

申请日：2020-04-10

Applicant: University of Massachusetts ,  Clinic for Special Children

Inventor： Guangping Gao ,  Huiya Yang ,  Kevin A. Strauss ,  Dan Wang

IPC: C12N9/10 ,  C12N15/86 ,  A61K48/00

Abstract: Aspects of the disclosure relate to compositions and methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof.

公开(公告)号：US11253576B2

公开(公告)日：2022-02-22

申请号：US15769981

申请日：2016-10-21

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Dominic Gessler

IPC: A61K38/50 ,  A61K48/00 ,  C12N15/00 ,  G01N33/50 ,  A61P25/28 ,  A61K9/00 ,  A61K45/06 ,  C12N7/00 ,  G01N33/66 ,  G01N33/68 ,  G01N33/84 ,  G01N33/06

Abstract: In some aspects, the disclosure relates to compositions and methods useful for the diagnosis and treatment of neurodegenerative diseases, such as leukodystrophies (e.g., Canavan Disease). In some embodiments, the methods comprise administering to a subject an N-acetylaspartate (NAA)-depleting agent or an N-acetylaspartate (NAA)-depleting agent based upon the subject's metabolic profile.