IN VIVO POLYNUCLEOTIDE DELIVERY CONJUGATES HAVING ENZYME SENSITIVE LINKAGES
The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active poiyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membr...
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| creator | ROZEMA, DAVID B CARLSON, JEFFREY C WAKEFIELD, DARREN H WOLFF, JON A MOHR, PETER HOFFMANN, TORSTEN JAHN-HOFMANN, KERSTIN KITAS, ERIC A BENSON, JONATHAN D HADWIGER, PHILIPP BLOKHIN, ANDREI V OTT, GUENTHER LEWIS, DAVID L MUELLER, HANS MARTIN ROEHL, INGO |
| description | The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active poiyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalentiy linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
La présente invention concerne des compositions pour l'administration de polynucléotides d'ARN interférence (ARNi) à des cellules in vivo. Les compositions comprennent des polyamines actives de membrane bipolaire modifiées de façon réversible avec des agents masquants de dipeptide-amidobenzyl-carbonate clivables par une enzyme. La modification masque l'activité membranaire du polymère tandis que la réversibilité produit une réponse physiologique. Les polyamines modifiées de façon réversible (polyconjugué dynamique ou DPC) sont en outre liés de façon covalente à un polynucléotide d'ARNi ou co-administrés avec un conjugué polynucléotide ARNi ciblé-molécule de ciblage. |
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| fullrecord | <record><control><sourceid>epo_EVB</sourceid><recordid>TN_cdi_epo_espacenet_CA2816041C</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>CA2816041C</sourcerecordid><originalsourceid>FETCH-epo_espacenet_CA2816041C3</originalsourceid><addsrcrecordid>eNrjZPD29FMI8wzzVwjw94n0C3X2cfUP8XRxVXBx9fEMcw2KVHD29_MKdXcMcQ1W8HAM8_RzV3D1i4r0dVUIdvUL9gwBKlLw8fTzdnR3DeZhYE1LzClO5YXS3Azybq4hzh66qQX58anFBYnJqXmpJfHOjkYWhmYGJobOxoRVAAAbRC1D</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>patent</recordtype></control><display><type>patent</type><title>IN VIVO POLYNUCLEOTIDE DELIVERY CONJUGATES HAVING ENZYME SENSITIVE LINKAGES</title><source>esp@cenet</source><creator>ROZEMA, DAVID B ; CARLSON, JEFFREY C ; WAKEFIELD, DARREN H ; WOLFF, JON A ; MOHR, PETER ; HOFFMANN, TORSTEN ; JAHN-HOFMANN, KERSTIN ; KITAS, ERIC A ; BENSON, JONATHAN D ; HADWIGER, PHILIPP ; BLOKHIN, ANDREI V ; OTT, GUENTHER ; LEWIS, DAVID L ; MUELLER, HANS MARTIN ; ROEHL, INGO</creator><creatorcontrib>ROZEMA, DAVID B ; CARLSON, JEFFREY C ; WAKEFIELD, DARREN H ; WOLFF, JON A ; MOHR, PETER ; HOFFMANN, TORSTEN ; JAHN-HOFMANN, KERSTIN ; KITAS, ERIC A ; BENSON, JONATHAN D ; HADWIGER, PHILIPP ; BLOKHIN, ANDREI V ; OTT, GUENTHER ; LEWIS, DAVID L ; MUELLER, HANS MARTIN ; ROEHL, INGO</creatorcontrib><description>The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active poiyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalentiy linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
La présente invention concerne des compositions pour l'administration de polynucléotides d'ARN interférence (ARNi) à des cellules in vivo. Les compositions comprennent des polyamines actives de membrane bipolaire modifiées de façon réversible avec des agents masquants de dipeptide-amidobenzyl-carbonate clivables par une enzyme. La modification masque l'activité membranaire du polymère tandis que la réversibilité produit une réponse physiologique. Les polyamines modifiées de façon réversible (polyconjugué dynamique ou DPC) sont en outre liés de façon covalente à un polynucléotide d'ARNi ou co-administrés avec un conjugué polynucléotide ARNi ciblé-molécule de ciblage.</description><language>eng ; fre</language><subject>ACYCLIC OR CARBOCYCLIC COMPOUNDS ; CHEMISTRY ; COMPOSITIONS BASED THEREON ; HUMAN NECESSITIES ; HYGIENE ; MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONSONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS ; MEDICAL OR VETERINARY SCIENCE ; METALLURGY ; ORGANIC CHEMISTRY ; ORGANIC MACROMOLECULAR COMPOUNDS ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES ; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS ; THEIR PREPARATION OR CHEMICAL WORKING-UP</subject><creationdate>2019</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=20190108&DB=EPODOC&CC=CA&NR=2816041C$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,776,881,25542,76290</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20190108&DB=EPODOC&CC=CA&NR=2816041C$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>ROZEMA, DAVID B</creatorcontrib><creatorcontrib>CARLSON, JEFFREY C</creatorcontrib><creatorcontrib>WAKEFIELD, DARREN H</creatorcontrib><creatorcontrib>WOLFF, JON A</creatorcontrib><creatorcontrib>MOHR, PETER</creatorcontrib><creatorcontrib>HOFFMANN, TORSTEN</creatorcontrib><creatorcontrib>JAHN-HOFMANN, KERSTIN</creatorcontrib><creatorcontrib>KITAS, ERIC A</creatorcontrib><creatorcontrib>BENSON, JONATHAN D</creatorcontrib><creatorcontrib>HADWIGER, PHILIPP</creatorcontrib><creatorcontrib>BLOKHIN, ANDREI V</creatorcontrib><creatorcontrib>OTT, GUENTHER</creatorcontrib><creatorcontrib>LEWIS, DAVID L</creatorcontrib><creatorcontrib>MUELLER, HANS MARTIN</creatorcontrib><creatorcontrib>ROEHL, INGO</creatorcontrib><title>IN VIVO POLYNUCLEOTIDE DELIVERY CONJUGATES HAVING ENZYME SENSITIVE LINKAGES</title><description>The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active poiyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalentiy linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
La présente invention concerne des compositions pour l'administration de polynucléotides d'ARN interférence (ARNi) à des cellules in vivo. Les compositions comprennent des polyamines actives de membrane bipolaire modifiées de façon réversible avec des agents masquants de dipeptide-amidobenzyl-carbonate clivables par une enzyme. La modification masque l'activité membranaire du polymère tandis que la réversibilité produit une réponse physiologique. Les polyamines modifiées de façon réversible (polyconjugué dynamique ou DPC) sont en outre liés de façon covalente à un polynucléotide d'ARNi ou co-administrés avec un conjugué polynucléotide ARNi ciblé-molécule de ciblage.</description><subject>ACYCLIC OR CARBOCYCLIC COMPOUNDS</subject><subject>CHEMISTRY</subject><subject>COMPOSITIONS BASED THEREON</subject><subject>HUMAN NECESSITIES</subject><subject>HYGIENE</subject><subject>MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONSONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS</subject><subject>MEDICAL OR VETERINARY SCIENCE</subject><subject>METALLURGY</subject><subject>ORGANIC CHEMISTRY</subject><subject>ORGANIC MACROMOLECULAR COMPOUNDS</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><subject>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</subject><subject>THEIR PREPARATION OR CHEMICAL WORKING-UP</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2019</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZPD29FMI8wzzVwjw94n0C3X2cfUP8XRxVXBx9fEMcw2KVHD29_MKdXcMcQ1W8HAM8_RzV3D1i4r0dVUIdvUL9gwBKlLw8fTzdnR3DeZhYE1LzClO5YXS3Azybq4hzh66qQX58anFBYnJqXmpJfHOjkYWhmYGJobOxoRVAAAbRC1D</recordid><startdate>20190108</startdate><enddate>20190108</enddate><creator>ROZEMA, DAVID B</creator><creator>CARLSON, JEFFREY C</creator><creator>WAKEFIELD, DARREN H</creator><creator>WOLFF, JON A</creator><creator>MOHR, PETER</creator><creator>HOFFMANN, TORSTEN</creator><creator>JAHN-HOFMANN, KERSTIN</creator><creator>KITAS, ERIC A</creator><creator>BENSON, JONATHAN D</creator><creator>HADWIGER, PHILIPP</creator><creator>BLOKHIN, ANDREI V</creator><creator>OTT, GUENTHER</creator><creator>LEWIS, DAVID L</creator><creator>MUELLER, HANS MARTIN</creator><creator>ROEHL, INGO</creator><scope>EVB</scope></search><sort><creationdate>20190108</creationdate><title>IN VIVO POLYNUCLEOTIDE DELIVERY CONJUGATES HAVING ENZYME SENSITIVE LINKAGES</title><author>ROZEMA, DAVID B ; CARLSON, JEFFREY C ; WAKEFIELD, DARREN H ; WOLFF, JON A ; MOHR, PETER ; HOFFMANN, TORSTEN ; JAHN-HOFMANN, KERSTIN ; KITAS, ERIC A ; BENSON, JONATHAN D ; HADWIGER, PHILIPP ; BLOKHIN, ANDREI V ; OTT, GUENTHER ; LEWIS, DAVID L ; MUELLER, HANS MARTIN ; ROEHL, INGO</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_CA2816041C3</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng ; fre</language><creationdate>2019</creationdate><topic>ACYCLIC OR CARBOCYCLIC COMPOUNDS</topic><topic>CHEMISTRY</topic><topic>COMPOSITIONS BASED THEREON</topic><topic>HUMAN NECESSITIES</topic><topic>HYGIENE</topic><topic>MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONSONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS</topic><topic>MEDICAL OR VETERINARY SCIENCE</topic><topic>METALLURGY</topic><topic>ORGANIC CHEMISTRY</topic><topic>ORGANIC MACROMOLECULAR COMPOUNDS</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><topic>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</topic><topic>THEIR PREPARATION OR CHEMICAL WORKING-UP</topic><toplevel>online_resources</toplevel><creatorcontrib>ROZEMA, DAVID B</creatorcontrib><creatorcontrib>CARLSON, JEFFREY C</creatorcontrib><creatorcontrib>WAKEFIELD, DARREN H</creatorcontrib><creatorcontrib>WOLFF, JON A</creatorcontrib><creatorcontrib>MOHR, PETER</creatorcontrib><creatorcontrib>HOFFMANN, TORSTEN</creatorcontrib><creatorcontrib>JAHN-HOFMANN, KERSTIN</creatorcontrib><creatorcontrib>KITAS, ERIC A</creatorcontrib><creatorcontrib>BENSON, JONATHAN D</creatorcontrib><creatorcontrib>HADWIGER, PHILIPP</creatorcontrib><creatorcontrib>BLOKHIN, ANDREI V</creatorcontrib><creatorcontrib>OTT, GUENTHER</creatorcontrib><creatorcontrib>LEWIS, DAVID L</creatorcontrib><creatorcontrib>MUELLER, HANS MARTIN</creatorcontrib><creatorcontrib>ROEHL, INGO</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>ROZEMA, DAVID B</au><au>CARLSON, JEFFREY C</au><au>WAKEFIELD, DARREN H</au><au>WOLFF, JON A</au><au>MOHR, PETER</au><au>HOFFMANN, TORSTEN</au><au>JAHN-HOFMANN, KERSTIN</au><au>KITAS, ERIC A</au><au>BENSON, JONATHAN D</au><au>HADWIGER, PHILIPP</au><au>BLOKHIN, ANDREI V</au><au>OTT, GUENTHER</au><au>LEWIS, DAVID L</au><au>MUELLER, HANS MARTIN</au><au>ROEHL, INGO</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>IN VIVO POLYNUCLEOTIDE DELIVERY CONJUGATES HAVING ENZYME SENSITIVE LINKAGES</title><date>2019-01-08</date><risdate>2019</risdate><abstract>The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active poiyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalentiy linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
La présente invention concerne des compositions pour l'administration de polynucléotides d'ARN interférence (ARNi) à des cellules in vivo. Les compositions comprennent des polyamines actives de membrane bipolaire modifiées de façon réversible avec des agents masquants de dipeptide-amidobenzyl-carbonate clivables par une enzyme. La modification masque l'activité membranaire du polymère tandis que la réversibilité produit une réponse physiologique. Les polyamines modifiées de façon réversible (polyconjugué dynamique ou DPC) sont en outre liés de façon covalente à un polynucléotide d'ARNi ou co-administrés avec un conjugué polynucléotide ARNi ciblé-molécule de ciblage.</abstract><oa>free_for_read</oa></addata></record> |
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| subjects | ACYCLIC OR CARBOCYCLIC COMPOUNDS CHEMISTRY COMPOSITIONS BASED THEREON HUMAN NECESSITIES HYGIENE MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONSONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS MEDICAL OR VETERINARY SCIENCE METALLURGY ORGANIC CHEMISTRY ORGANIC MACROMOLECULAR COMPOUNDS PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS THEIR PREPARATION OR CHEMICAL WORKING-UP |
| title | IN VIVO POLYNUCLEOTIDE DELIVERY CONJUGATES HAVING ENZYME SENSITIVE LINKAGES |
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