SINGLE CHAIN VARIABLE FRAGMENT (SCFV) MODIFIED LIPID NANOPARTICLE COMPOSITIONS AND USES THEREOF

Provided herein are pharmaceutical compositions comprising a lipid nanoparticle (LNP) and a therapeutic nucleic acid (TNA), wherein the LNP comprises a single-chain variable fragment (scFv) linked to the LNP, and at least one pharmaceutically acceptable excipient. The scFv is capable of binding an a...

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Bibliographische Detailangaben
Hauptverfasser:	NOLTING Birte, SILVER Nathaniel, LI Prudence Yui Tung, TOY Randall Newton, OONTHONPAN Lalita, SAMAYOA Phillip
Format:	Patent
Sprache:	eng ; spa
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Provided herein are pharmaceutical compositions comprising a lipid nanoparticle (LNP) and a therapeutic nucleic acid (TNA), wherein the LNP comprises a single-chain variable fragment (scFv) linked to the LNP, and at least one pharmaceutically acceptable excipient. The scFv is capable of binding an antigen present on the surface of a cell, advantageously providing LNP compositions that target only those cells or tissues expressing the receptor. En la presente descripción se proporcionan composiciones farmacéuticas que comprenden una nanopartícula lipídica (LNP) y un ácido nucleico terapéutico (TNA), en donde la LNP comprende un fragmento variable monocatenario (scFv) unido a la LNP, y al menos un excipiente farmacéuticamente aceptable. El scFv es capaz de unirse a un antígeno presente en la superficie de una célula, proporcionando ventajosamente composiciones de LNP que se dirigen solo a aquellas células o tejidos que expresan el receptor.