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1.发明公开公开(公告)号：US20240254462A1
公开(公告)日：2024-08-01
申请号：US18405286
申请日：2024-01-05
发明人： Laura Anh Nguyen , Jeremiah D. Farelli , Adam Walter Scheidegger , Abigail Elizabeth Witt , Justin Chen , James Scott Sullivan
CPC分类号： C12N9/22 , A61K9/5123 , C12N9/1007 , C12N15/113 , C12N15/86 , A61K38/00 , C07K2319/81 , C12N2310/20 , C12Y305/01098
摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a secreted frizzled related protein 1 (SFRP1) gene by targeting an SFRP1 expression control region and methods of use thereof for treating an SFRP1 associated disorder, e.g., hair loss.
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公开(公告)号：US20220348908A1
公开(公告)日：2022-11-03
申请号：US17029820
申请日：2020-09-23
发明人： Jesse Jerome Smith , Jodi Michelle Kennedy , Jeremiah D. Farelli , Kendrick Alan Goss , Adam Walter Scheidegger , Yoseph Kassa , Christian Wessel Cobaugh , Timsi Rao
摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a hepatocyte nuclear factor 4 alpha (HNF4a) gene by targeting an HNF4α expression control region and methods of use thereof for treating an HNF4α associated disorder, e.g., cirrhosis.
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3.发明授权公开(公告)号：US11987791B2
公开(公告)日：2024-05-21
申请号：US17029820
申请日：2020-09-23
发明人： Jesse Jerome Smith , Jodi Michelle Kennedy , Jeremiah D. Farelli , Kendrick Alan Goss , Adam Walter Scheidegger , Yoseph Kassa , Christian Wessel Cobaugh , Timsi Rao
CPC分类号： C12N15/11 , C12N9/22 , C07K2319/80 , C07K2319/81 , C12N2310/20
摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a hepatocyte nuclear factor 4 alpha (HNF4α) gene by targeting an HNF4α expression control region and methods of use thereof for treating an HNF4α associated disorder, e.g., cirrhosis.
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4.发明公开公开(公告)号：US20240115730A1
公开(公告)日：2024-04-11
申请号：US18255906
申请日：2021-12-15
CPC分类号： A61K47/6929 , A61K47/543 , A61K47/60
摘要： The instant disclosure relates to nucleic acid-lipid particles having 1,2-dioleoyl-3-trimethylammonium-propane (DOTAP), which preferentially localize and deliver associated cargoes to the lung and various lung tissues, as well as to tissues to which such particles are directly injected. The instant disclosure provides compositions comprising such lipid particles, optionally in association with a therapeutic agent (e.g., a therapeutic mRNA and/or nucleic acid controller system), as well as methods and kits for delivering a lipid particle-associated therapeutic agent and/or treating a disease or disorder, e.g., a lung disease or disorder, in a subject, using the lipid particle compositions provided herein.
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5.发明申请公开(公告)号：US20220348893A1
公开(公告)日：2022-11-03
申请号：US17754051
申请日：2020-09-23
发明人： Serena Lunardi , Adam Walter Scheidegger , Jesse Jerome Smith , Jeremiah Dale Farelli , Jodi Michelle Kennedy
摘要： The present disclosure relates generally to methods and compositions for modulating frataxin (FXN) expression, e.g., to treat Friedreich ataxia (FRDA).
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公开(公告)号：US20220288237A1
公开(公告)日：2022-09-15
申请号：US17701325
申请日：2022-03-22
发明人： Vishwesh Ashok Patil , Jeremiah D. Farelli , Rahul Karnik , Jesse Jerome Smith , Can Sarisozen , Adam Walter Scheidegger , Barbara Bennett
IPC分类号： A61K48/00 , C12N15/63 , A61K31/7115 , A61K9/127
摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of an apolipoprotein B (APOB) gene by targeting an APOB expression control region and methods of use thereof for treating an APOB associated disorder, e.g., hypercholesterolemia.
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7.发明公开公开(公告)号：US20240344061A1
公开(公告)日：2024-10-17
申请号：US18637630
申请日：2024-04-17
发明人： Jesse Jerome Smith , Jodi Michelle Kennedy , Jeremiah D. Farelli , Kendrick Alan Goss , Adam Walter Scheidegger , Yoseph Kassa , Christian Wessel Cobaugh , Timsi Rao
CPC分类号： C12N15/11 , C12N9/22 , C07K2319/80 , C07K2319/81 , C12N2310/20
摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a hepatocyte nuclear factor 4 alpha (HNF4α) gene by targeting an HNF4α expression control region and methods of use thereof for treating an HNF4α associated disorder, e.g., cirrhosis.
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公开(公告)号：US20230114151A1
公开(公告)日：2023-04-13
申请号：US17929828
申请日：2022-09-06
发明人： Timsi Rao , Jodi Kennedy , Pankaj Mandal , Jeremiah D. Farelli
摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a forkhead box P3 (FOXP3) gene by targeting a FOXP3 expression control region and methods of use thereof for treating a FOXP3 associated disorder, such as an autoimmune disease, e.g., IPEX syndrome.
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公开(公告)号：US20240197636A1
公开(公告)日：2024-06-20
申请号：US18556563
申请日：2022-04-21
IPC分类号： A61K9/16 , A61K47/54 , A61P1/16 , C12N15/113
CPC分类号： A61K9/1617 , A61K47/543 , A61P1/16 , C12N15/113 , C12N2310/14
摘要： The instant disclosure relates to nucleic acid-lipid particles that mix ionizable and cationic lipids, which preferentially localize and deliver associated cargoes to the liver when administered parenterally, and which optionally can be used to deliver associated cargoes to tissues to which such particles are directly injected. The instant disclosure provides compositions comprising such lipid particles, optionally in association with a therapeutic agent (e.g., a therapeutic mRNA and/or nucleic acid controller system), as well as methods and kits for delivering a lipid particle-associated therapeutic agent and/or treating a disease or disorder, e.g., a liver disease or disorder, in a subject, using the lipid particle compositions provided herein.
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