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公开(公告)号：US20240173420A1
公开(公告)日：2024-05-30
申请号：US18375206
申请日：2023-09-29
发明人： Claudio Punzo , Anastasia Khvorova , Dimas Echeverria Moreno , Annabelle Biscans , Julia F. Alterman , Matthew Hassler , Shun-Yun Cheng
CPC分类号： A61K47/554 , A61K9/0048 , A61K47/542 , A61K47/543 , A61K47/545 , A61K47/548
摘要： Provided herein are conjugated oligonucleotides that are characterized by efficient and specific eye distribution.
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公开(公告)号：US20230340529A1
公开(公告)日：2023-10-26
申请号：US18024359
申请日：2021-09-02
发明人： Guangping Gao , Philip Tai , Claudio Punzo , Haijiang Lin
IPC分类号： C12N15/86 , A61K35/761
CPC分类号： C12N15/86 , A61K35/761 , C12N2750/14143 , C12N2750/14171 , C12N2750/14122
摘要： 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.
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公开(公告)号：US20240182904A1
公开(公告)日：2024-06-06
申请号：US18375235
申请日：2023-09-29
发明人： Claudio Punzo , Anastasia Khvorova , Dimas Echeverria Moreno , Annabelle Biscans , Julia F. Alterman , Matthew Hassler , Shun-Yun Cheng , Jillian Caiazzi
IPC分类号： C12N15/113 , A61K47/54 , A61P27/02 , C12N15/86
CPC分类号： C12N15/1137 , A61K47/549 , A61P27/02 , C12N15/86 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2750/14122 , C12N2750/14143
摘要： Provided herein are oligonucleotides (e.g., siRNA) targeting S6K1. Also provided are methods of treating a disease associated with S6K1 expression.
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公开(公告)号：US20230048017A1
公开(公告)日：2023-02-16
申请号：US17685457
申请日：2022-03-03
发明人： Guangping Gao , Phillip Tai , Claudio Punzo , Haijiang Lin
IPC分类号： C12N15/86 , C07K14/005 , C12N7/00 , A61K35/76 , A61P9/00
摘要： 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.
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公开(公告)号：US20230220395A1
公开(公告)日：2023-07-13
申请号：US17996711
申请日：2021-04-20
发明人： Claudio Punzo , Shun-Yun Cheng
IPC分类号： C12N15/113 , A61K31/506 , A61K31/216 , A61K31/42 , A61K31/202 , A61P27/02
CPC分类号： C12N15/1137 , A61K31/506 , A61K31/216 , A61K31/42 , A61K31/202 , A61P27/02
摘要： Aspects of the disclosure relate to methods and compositions for treatment of certain ocular diseases and disorders, for example age-related macular degeneration (AMD). In some embodiments, the methods comprise administering a subject having AMD one or more therapeutic agents that modulate the mTORCl pathway (or a component thereof). The disclosure is based, in part, on methods for treating AMD in a subject by administering one or more kinase inhibitors, for example one or more serine/threonine kinase inhibitors. In some embodiments, at least one of the serine/threonine kinase inhibitors is a Ribosomal protein S6 kinase beta-1 (S6K1) inhibitor.
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公开(公告)号：US20230057380A1
公开(公告)日：2023-02-23
申请号：US17779793
申请日：2020-11-25
发明人： Guangping Gao , Phillip Tai , Claudio Punzo , Zunhong Ke
摘要： Aspects of the disclosure relate to a recombinant adeno-virus encoding an anti-Vascular endothelial cell growth factor (VEGF) agent in a cell or subject (e.g., rAAV2.7m8-KH902). In some embodiments, compositions described herein are useful for treating subjects having diseases associated with angiogenesis or aberrant VEGF activity/signaling.
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7.发明申请公开(公告)号：US20220332792A1
公开(公告)日：2022-10-20
申请号：US17639962
申请日：2020-09-03
发明人： Guangping Gao , Phillip Tai , Claudio Punzo
摘要： Aspects of the disclosure relate to compositions and methods for expressing one or more anti-Vascular endothelial cell growth factor (VEGF) agents in a cell or subject. In some embodiments, the disclosure provides isolated nucleic acids and rAAVs comprising 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.
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