Invention Publication
- Patent Title: EXTRAHEPATIC DELIVERY
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Application No.: US18311484Application Date: 2023-05-03
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Publication No.: US20240009225A1Publication Date: 2024-01-11
- Inventor: Jayaprakash K. NAIR , Martin MAIER , Vasant JADHAV , Stuart MILSTEIN , Kirk BROWN , Rubina G. PARMAR , Kallanthottathil G. RAJEEV , Muthiah MANOHARAN , Alexander V. KEL'IN , Muthusamy JAYARAMAN , Klaus CHARISSE , Adam CASTORENO , Christopher THEILE , Kevin FITZGERALD
- Applicant: ALNYLAM PHARMACEUTICALS, INC.
- Applicant Address: US MA Cambridge
- Assignee: ALNYLAM PHARMACEUTICALS, INC.
- Current Assignee: ALNYLAM PHARMACEUTICALS, INC.
- Current Assignee Address: US MA Cambridge
- Main IPC: A61K31/713
- IPC: A61K31/713 ; A61K47/54 ; A61K9/00 ; C12N15/113

Abstract:
One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.
Public/Granted literature
- US12005074B2 Extrahepatic delivery Public/Granted day:2024-06-11
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