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公开(公告)号：WO2023064781A1
公开(公告)日：2023-04-20
申请号：PCT/US2022/077923
申请日：2022-10-11
发明人： YOZWIAK, Nathan, Lawrence , ARZE, Cesar, A. , NAWANDAR, Dhananjay Maniklal , DELAGRAVE, Simon , HAJJAR, Roger, Joseph , ONG, Tuyen
摘要： This invention relates generally to Anelloviridae family vectors (e.g., anellovectors) and compositions and uses thereof.
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公开(公告)号：WO2022140560A1
公开(公告)日：2022-06-30
申请号：PCT/US2021/064887
申请日：2021-12-22
摘要： This invention relates generally to compositions for making anellovectors and uses thereof.
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公开(公告)号：WO2021252955A1
公开(公告)日：2021-12-16
申请号：PCT/US2021/037091
申请日：2021-06-11
IPC分类号： C12N15/86 , C12N7/00 , A61K48/00 , A61P31/00 , C12N2750/00021 , C12N2750/00043 , C12N2750/00044
摘要： This invention relates generally to compositions for making anellovectors and uses thereof. For instance, a method herein can comprise providing a nucleic acid construct that comprises a first Anellovirus genome encoding an exogenous effector and a second Anellovirus genome or fragment thereof, arranged in tandem. In some embodiments, the nucleic acid construct results in production of an anellovector comprising an Anellovirus genetic element encoding the exogenous effector, enclosed in a proteinaceous exterior.
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4.发明申请
公开(公告)号：WO2021113635A1
公开(公告)日：2021-06-10
申请号：PCT/US2020/063307
申请日：2020-12-04
发明人： CASEY, John, Patrick, Jr. , BERRY, David, Arthur , BRIGGS, Timothy, F. , BUCKBINDER, Leonard , KIM, Mi-Jeong , LIANG, Anna , PANDIAN, Anushya
IPC分类号： A61K31/035 , A61K31/19 , A61K31/616 , C07C57/42 , C07C57/44 , C07C69/017
摘要： Disclosed herein are acylated active agents, compositions containing them, unit dosage forms containing them, and methods of their use, e.g., for treating a metabolic disorder or nonalcoholic fatty liver disease or for modulating a metabolic marker or nonalcoholic fatty liver disease marker.
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公开(公告)号：WO2020118178A8
公开(公告)日：2020-06-11
申请号：PCT/US2019/064926
申请日：2019-12-06
发明人： CASEY, John, Patrick, Jr. , BERRY, David, Arthur , ALEXANDER, Jessica, Elizabeth , BRIGGS, Timothy , BUCKBINDER, Leonard , GUNASEKERA, Dinara, Shashanka , KAMALI SARVESTANI, Afrand , KIM, Mi-Jeong , LANTER, Bernard , LIANG, Anna , NUDEL, Kathleen , PECK, Spencer, Cory , ROSS, Cheri , TAYLOR, Steven, John , YASUDA, Koji
IPC分类号： A61K31/225 , A61K47/54 , A61P25/00 , A61P37/02
摘要： Disclosed are conjugates of monomethyl fumarate and a carrier group or aminocarrier group, or a pharmaceutically acceptable salt thereof. In the conjugates, monomethyl fumarate acyl is covalently bonded to the carrier group or aminocarrier group through a carbon-oxygen bond that is cleavable in vivo. The carrier group may include a core, e.g., a monosaccharide, a sugar acid (e.g., acid monosaccharide), a sugar alcohol, or a catechin polyphenol. The aminocarrier group may include a core, e.g., an aminomonosaccharide. The carrier group or aminocarrier group may include, e.g., at least one short chain fatty acid acyl, at least one tryptophan analogue, at least one ketone body, or at least one preketone body. Also disclosed are pharmaceutical compositions containing the conjugates and methods of their use.
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公开(公告)号：WO2020102503A3
公开(公告)日：2020-05-22
申请号：PCT/US2019/061434
申请日：2019-11-14
发明人： VON MALTZAHN, Geoffrey, A. , RUBENS, Jacob, Rosenblum , SHAH, Jagesh, Vijaykumar , RUZO MATIAS, Albert , PUCCI, Ferdinando , MILWID, John, Miles , MEE, Michael, Travis , GORDON, Neal, Francis
摘要： The present disclosure provides, at least in part, methods and compositions for in vivo fusosome delivery. In some embodiments, the fusosome comprises a combination of elements that promote specificity for target cells, e.g., one or more of a fusogen, a positive target cell- specific regulatory element, and a non-target cell- specific regulatory element. In some embodiments, the fusosome comprises one or more modifications that decrease an immune response against the fusosome.
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公开(公告)号：WO2020102503A2
公开(公告)日：2020-05-22
申请号：PCT/US2019/061434
申请日：2019-11-14
发明人： VON MALTZAHN, Geoffrey, A. , RUBENS, Jacob, Rosenblum , SHAH, Jahesh, Vijaykumar , RUZO MATIAS, Albert , PUCCI, Ferdinando , MILWID, John, Miles , MEE, Michael, Travis , GORDON, Neal, Francis
摘要： The present disclosure provides, at least in part, methods and compositions for in vivo fusosome delivery. In some embodiments, the fusosome comprises a combination of elements that promote specificity for target cells, e.g., one or more of a fusogen, a positive target cell- specific regulatory element, and a non-target cell- specific regulatory element. In some embodiments, the fusosome comprises one or more modifications that decrease an immune response against the fusosome.
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公开(公告)号：WO2020102499A3
公开(公告)日：2020-05-22
申请号：PCT/US2019/061424
申请日：2019-11-14
发明人： VON MALTZAHN, Geoffrey, A. , RUBENS, Jacob, Rosenblum , SHAH, Jagesh, Vijaykumar , RUZO MATIAS, Albert , PUCCI, Ferdinando , MILWID, John, Miles , MEE, Michael, Travis , GORDON, Neal, Francis
摘要： The present disclosure provides, at least in part, methods and compositions for in vivo fusosome delivery. In some embodiments, the fusosome comprises a combination of elements that promote specificity for target cells, e.g., one or more of a fusogen, a positive target cell- specific regulatory element, and a non-target cell- specific regulatory element. In some embodiments, the fusosome comprises one or more modifications that decrease an immune response against the fusosome.
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公开(公告)号：WO2020102499A2
公开(公告)日：2020-05-22
申请号：PCT/US2019/061424
申请日：2019-11-14
发明人： VON MALTZAHN, Geoffrey, A. , RUBENS, Jacob, Rosenblum , SHAH, Jagesh, Vijaykumar , RUZO MATIAS, Albert , PUCCI, Ferdinando , MILWID, John, Miles , MEE, Michael, Travis , GORDON, Neal, Francis
摘要： The present disclosure provides, at least in part, methods and compositions for in vivo fusosome delivery. In some embodiments, the fusosome comprises a combination of elements that promote specificity for target cells, e.g., one or more of a fusogen, a positive target cell- specific regulatory element, and a non-target cell- specific regulatory element. In some embodiments, the fusosome comprises one or more modifications that decrease an immune response against the fusosome.
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公开(公告)号：WO2019241746A1
公开(公告)日：2019-12-19
申请号：PCT/US2019/037373
申请日：2019-06-14
IPC分类号： A61K31/222 , A61K31/4409 , A61P1/04 , A61P3/10 , A61P9/10 , A61P11/06 , A61P17/06 , A61P19/02 , A61P25/00 , A61P29/00 , A61P37/06 , A61P37/08 , A61P43/00 , G01N33/00
摘要： The invention provides methods of decreasing immune response by inhibiting iron-dependent cellular disassembly. The decrease in immune response may be used, for example, for treatment of a disorder associated with iron-dependent cellular disassembly, including an autoimmune disorder, allergy, or an inflammatory disorder. The invention also provides screening assays for identification of compounds that inhibit iron-dependent cellular disassembly and are also immunoinhibitory agents.
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