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公开(公告)号：US20240254430A1
公开(公告)日：2024-08-01
申请号：US17050608
申请日：2019-04-27
申请人： REGENXBIO INC.
发明人： Claire G. ZHANG , Shaojie WENG , Ya-Chen CHANG , Franz M. GERNER
CPC分类号： C12M47/02 , B01D39/18 , B01D39/2068 , C12N15/86 , B01D2239/065 , C12N2750/14143 , C12N2750/14151
摘要： Provided herein are scalable methods for the clarification of a composition comprising recombinant Adeno-Associated Virus (rAAV) particles and an impurity.
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公开(公告)号：US20230390418A1
公开(公告)日：2023-12-07
申请号：US18034334
申请日：2021-10-29
申请人： REGENXBIO INC.
发明人： Joseph Bruder , Ye Liu
CPC分类号： A61K48/0058 , C12N15/86 , A61K48/0041 , A61K48/0066 , C07K16/36 , C07K2317/20 , C12N2750/14143 , C12N2750/14151 , C12N2750/14171 , C12N2750/14122 , C12N2830/15 , A61P7/10
摘要： Compositions and methods are described for the delivery of a fully human post-translationally modified therapeutic monoclonal antibody that binds to factor XII to a human subject diagnosed with a disease or condition indicated for treatment with an anti-factor XII antibody. Such diseases include hereditary angioedema, as well as thrombosis and hypercoagulation.
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公开(公告)号：US20230374541A1
公开(公告)日：2023-11-23
申请号：US18030683
申请日：2021-10-07
申请人： REGENXBIO INC.
发明人： Olivier Danos , Samantha Yost , Andrew Mercer , Ye Liu , Joseph Bruder , Subha Karumuthil-Melethil , Elad Firnberg , Randolph Qian , April R. Tepe , Jennifer M. Egley
CPC分类号： C12N15/86 , A61K48/0058 , C12N2750/14122 , C12N2750/14143
摘要： The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins engineered to include amino acid sequences and/or amino acid substitutions that confer and/or enhance desired properties, particularly increased transduction in CNS or muscle cells relative to a rAAV having a reference capsid.
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4.发明公开公开(公告)号：US20230372538A1
公开(公告)日：2023-11-23
申请号：US18030621
申请日：2021-10-06
申请人： REGENXBIO Inc.
CPC分类号： A61K48/0041 , A61K9/0048 , C07K16/22 , A61K47/26 , A61P27/02 , C12N15/86 , A61K2039/505
摘要： Provided herein are pharmaceutical compositions for administration to a suprachoroidal space of an eye of a subject. The pharmaceutical compositions can include a recombinant adeno-associated virus (AAV) encoding a transgene. Also provided herein are methods for treating or preventing a disease in a subject by administering a therapeutically effective amount of the pharmaceutical compositions to the subject in need.
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公开(公告)号：US20230364206A1
公开(公告)日：2023-11-16
申请号：US18030613
申请日：2021-10-06
申请人： REGENXBIO Inc.
发明人： Christina Maria Ohnsman , Stephen Joseph Pakola , Sherri Van Everen , Paulo Falabella , Alexander Marston Bailey , Nicholas Alexander Piers Sascha Buss , Kwi Hye Kim
CPC分类号： A61K38/4813 , C12N9/485 , A61K48/0066 , C12N15/86 , C12Y304/14009 , A61P27/02 , A61K48/0075 , C12N2750/14143
摘要： Compositions and methods are described for the delivery of therapeutic products (such as therapeutic proteins (for example, antibodies), therapeutic RNAs (for example, shRNAs, siRNAs, and miRNAs), and therapeutic aptamers) to the retina/vitreal humour in the eyes of human subjects to treat pathologies of the eye, involving, for example, recombinant viral vectors such as recombinant adeno-associated virus (rAAV) vectors.
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6.发明申请公开(公告)号：US20220211875A1
公开(公告)日：2022-07-07
申请号：US17574118
申请日：2022-01-12
申请人： Regents of the University of Minnesota , HealthPartners Institute f/k/a HealthPartners Research Foundation , Regenxbio Inc.
IPC分类号： A61K48/00 , A61K31/675 , A61K38/47 , A61K45/06 , C12N15/86 , A61P25/28 , A61K9/00 , C12N7/00
摘要： A method to prevent, inhibit or treat one or more symptoms associated with disease of the central nervous system by intranasally, intrathecally, intracerebrovascularly or intravenously
administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease, in an amount effective, e.g., to provide for cross-correction.-
公开(公告)号：US20220143221A1
公开(公告)日：2022-05-12
申请号：US17600377
申请日：2020-04-02
申请人： REGENXBIO, Inc.
发明人： Olivier Danos , Sherri Van Everen , Jesse I. Yoo , Samir Maganbhai Patel , Avanti Arvind Ghanekar , Anthony Ray O'Berry , Kim Rees Irwin-Pack , Darin Thomas Curtiss
摘要： Compositions and methods are described for the delivery of therapeutic products (such as therapeutic proteins (for example, antibodies), therapeutic RNAs (for example, shRNAs, siRNAs, and miRNAs), and therapeutic aptamers) to the retina/vitreal humour in the eyes of human subjects to treat pathologies of the eye, involving, for example, recombinant viral vectors such as recombinant adeno-associated virus (rAAV) vectors.
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公开(公告)号：US20210346473A1
公开(公告)日：2021-11-11
申请号：US17212516
申请日：2021-03-25
摘要： A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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公开(公告)号：US20210163991A1
公开(公告)日：2021-06-03
申请号：US17267247
申请日：2019-08-09
申请人： REGENXBIO Inc.
发明人： Michael GILLMEISTER , Miguel VALLE , Robert STADELMAN , Franz GERNER , Matthieu GUIBERT , Bhargavi KONDRAGUNTA
摘要： Provided herein are improved methods for producing recombinant Adeno-Associated Virus (rAAV) particles. In some embodiments, a method for producing recombinant AAV (rAAV) particles provided herein comprises culturing cells capable of producing rAAV particles in the presence of a histone deacetylase (HDAC) inhibitor. In some embodiments, a method for producing recombinant AAV (rAAV) particles provided herein comprises culturing cells capable of producing rAAV particles in the presence of a histone deacetylase (HDAC) inhibitor and increased amount of a sodium salt.
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公开(公告)号：US20210085759A1
公开(公告)日：2021-03-25
申请号：US17118353
申请日：2020-12-10
摘要： A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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