GENE EDITING OF PCSK9 OR ANGPTL3 AND COMPOSITIONS AND METHODS OF USING SAME FOR TREATMENT OF DISEASE

Disclosed herein are novel gene editing systems capable of being delivered to a subject intravenously through a lipid nano particle pharmaceutical formulation and producing durable in vivo editing of a target gene, such as ANGPTL3, with high on-target gene editing efficiency, reduced or low off-targ...

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Bibliographic Details
Main Authors CHENG, Christopher, REISS, Caroline, LEE, Richard, Glenn, DE ALMEIDA PINTO GARCIA, Sara, Cristina, BELLINGER, Andrew, M, RAJEEV, Kallanthottathil, G, JAYARAM, Hariharan, CHADWICK, Alexandra, WANG, Kui, ROHDE, Ellen, IYER, Sowmya, DENIZIO, Jamie
Format Patent
LanguageEnglish
French
German
Published 31.07.2024
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
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Summary:Disclosed herein are novel gene editing systems capable of being delivered to a subject intravenously through a lipid nano particle pharmaceutical formulation and producing durable in vivo editing of a target gene, such as ANGPTL3, with high on-target gene editing efficiency, reduced or low off-target effect, and no germline editing. The gene editing systems comprise a chemically modified guide nucleic acid sequence with a spacer having a specified arrangement of deoxyribonucleotides and ribonucleotides. The novel gene editing systems comprise mRNA that encodes the gene editor proteins, which may include a modified nickase component. Methods of disease treatments using the gene editing systems are also disclosed.
Bibliography:Application Number: EP20220873600