Ionizable lipids and nanoparticle compositions thereof
Provided herein are ionizable lipids represented by Formula (I): or a pharmaceutically acceptable salt thereof, wherein R1, R2, R3, R4, R5, R6, R1 ', R2', R3 ', R4', R5 ', R6', m, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) composition...
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| creator | LEBLANC MICHAEL CHATTERTON, JON, E STANTON MATTHEW G NOLTING, BJU RN FEINSTEIN, GUY |
| description | Provided herein are ionizable lipids represented by Formula (I): or a pharmaceutically acceptable salt thereof, wherein R1, R2, R3, R4, R5, R6, R1 ', R2', R3 ', R4', R5 ', R6', m, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising an ionizable lipid of the invention and a capsid-free non-viral vector (e.g., ceDNA). These LNPs can be used to deliver capsid-free non-viral DNA vectors to target sites of interest (e.g., cells, tissues, organs, etc.).
本文提供了由式(I)表示的可电离脂质：或其药学上可接受的盐，其中R1、R2、R3、R4、R5、R6、R1'、R2'、R3'、R4'、R5'、R6'、m和n如本文所定义。本文还提供了脂质纳米颗粒(LNP)组合物，其包含本发明的可电离脂质和无衣壳的非病毒载体(例如ceDNA)。这些LNP可用于将无衣壳的非病毒DNA载体递送至所关注的靶位点(例如，细胞、组织、器官等)。 |
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本文提供了由式(I)表示的可电离脂质：或其药学上可接受的盐，其中R1、R2、R3、R4、R5、R6、R1'、R2'、R3'、R4'、R5'、R6'、m和n如本文所定义。本文还提供了脂质纳米颗粒(LNP)组合物，其包含本发明的可电离脂质和无衣壳的非病毒载体(例如ceDNA)。这些LNP可用于将无衣壳的非病毒DNA载体递送至所关注的靶位点(例如，细胞、组织、器官等)。</description><language>chi ; eng</language><subject>ACYCLIC OR CARBOCYCLIC COMPOUNDS ; CHEMISTRY ; HETEROCYCLIC COMPOUNDS ; HUMAN NECESSITIES ; HYGIENE ; MEDICAL OR VETERINARY SCIENCE ; METALLURGY ; ORGANIC CHEMISTRY ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES ; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</subject><creationdate>2022</creationdate><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20220722&DB=EPODOC&CC=CN&NR=114787127A$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,780,885,25564,76547</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20220722&DB=EPODOC&CC=CN&NR=114787127A$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>LEBLANC MICHAEL</creatorcontrib><creatorcontrib>CHATTERTON, JON, E</creatorcontrib><creatorcontrib>STANTON MATTHEW G</creatorcontrib><creatorcontrib>NOLTING, BJU RN</creatorcontrib><creatorcontrib>FEINSTEIN, GUY</creatorcontrib><title>Ionizable lipids and nanoparticle compositions thereof</title><description>Provided herein are ionizable lipids represented by Formula (I): or a pharmaceutically acceptable salt thereof, wherein R1, R2, R3, R4, R5, R6, R1 ', R2', R3 ', R4', R5 ', R6', m, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising an ionizable lipid of the invention and a capsid-free non-viral vector (e.g., ceDNA). These LNPs can be used to deliver capsid-free non-viral DNA vectors to target sites of interest (e.g., cells, tissues, organs, etc.).
本文提供了由式(I)表示的可电离脂质：或其药学上可接受的盐，其中R1、R2、R3、R4、R5、R6、R1'、R2'、R3'、R4'、R5'、R6'、m和n如本文所定义。本文还提供了脂质纳米颗粒(LNP)组合物，其包含本发明的可电离脂质和无衣壳的非病毒载体(例如ceDNA)。这些LNP可用于将无衣壳的非病毒DNA载体递送至所关注的靶位点(例如，细胞、组织、器官等)。</description><subject>ACYCLIC OR CARBOCYCLIC COMPOUNDS</subject><subject>CHEMISTRY</subject><subject>HETEROCYCLIC COMPOUNDS</subject><subject>HUMAN NECESSITIES</subject><subject>HYGIENE</subject><subject>MEDICAL OR VETERINARY SCIENCE</subject><subject>METALLURGY</subject><subject>ORGANIC CHEMISTRY</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><subject>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2022</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZDDzzM_LrEpMyklVyMksyEwpVkjMS1HIS8zLL0gsKslMBoon5-cW5BdnlmTm5xUrlGSkFqXmp_EwsKYl5hSn8kJpbgZFN9cQZw_d1IL8-NTigsTk1LzUknhnP0NDE3MLc0Mjc0djYtQAAN3dLjI</recordid><startdate>20220722</startdate><enddate>20220722</enddate><creator>LEBLANC MICHAEL</creator><creator>CHATTERTON, JON, E</creator><creator>STANTON MATTHEW G</creator><creator>NOLTING, BJU RN</creator><creator>FEINSTEIN, GUY</creator><scope>EVB</scope></search><sort><creationdate>20220722</creationdate><title>Ionizable lipids and nanoparticle compositions thereof</title><author>LEBLANC MICHAEL ; CHATTERTON, JON, E ; STANTON MATTHEW G ; NOLTING, BJU RN ; FEINSTEIN, GUY</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_CN114787127A3</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>chi ; eng</language><creationdate>2022</creationdate><topic>ACYCLIC OR CARBOCYCLIC COMPOUNDS</topic><topic>CHEMISTRY</topic><topic>HETEROCYCLIC COMPOUNDS</topic><topic>HUMAN NECESSITIES</topic><topic>HYGIENE</topic><topic>MEDICAL OR VETERINARY SCIENCE</topic><topic>METALLURGY</topic><topic>ORGANIC CHEMISTRY</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><topic>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</topic><toplevel>online_resources</toplevel><creatorcontrib>LEBLANC MICHAEL</creatorcontrib><creatorcontrib>CHATTERTON, JON, E</creatorcontrib><creatorcontrib>STANTON MATTHEW G</creatorcontrib><creatorcontrib>NOLTING, BJU RN</creatorcontrib><creatorcontrib>FEINSTEIN, GUY</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>LEBLANC MICHAEL</au><au>CHATTERTON, JON, E</au><au>STANTON MATTHEW G</au><au>NOLTING, BJU RN</au><au>FEINSTEIN, GUY</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>Ionizable lipids and nanoparticle compositions thereof</title><date>2022-07-22</date><risdate>2022</risdate><abstract>Provided herein are ionizable lipids represented by Formula (I): or a pharmaceutically acceptable salt thereof, wherein R1, R2, R3, R4, R5, R6, R1 ', R2', R3 ', R4', R5 ', R6', m, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising an ionizable lipid of the invention and a capsid-free non-viral vector (e.g., ceDNA). These LNPs can be used to deliver capsid-free non-viral DNA vectors to target sites of interest (e.g., cells, tissues, organs, etc.).
本文提供了由式(I)表示的可电离脂质：或其药学上可接受的盐，其中R1、R2、R3、R4、R5、R6、R1'、R2'、R3'、R4'、R5'、R6'、m和n如本文所定义。本文还提供了脂质纳米颗粒(LNP)组合物，其包含本发明的可电离脂质和无衣壳的非病毒载体(例如ceDNA)。这些LNP可用于将无衣壳的非病毒DNA载体递送至所关注的靶位点(例如，细胞、组织、器官等)。</abstract><oa>free_for_read</oa></addata></record> |
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| subjects | ACYCLIC OR CARBOCYCLIC COMPOUNDS CHEMISTRY HETEROCYCLIC COMPOUNDS HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY ORGANIC CHEMISTRY PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS |
| title | Ionizable lipids and nanoparticle compositions thereof |
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