Invention Grant
US09402816B2 Diester and triester based low molecular weight, biodegradeable cationic lipids for oligonucleotide delivery
有权
用于寡核苷酸递送的基于二酯和三酯的低分子量生物降解阳离子脂质
- Patent Title: Diester and triester based low molecular weight, biodegradeable cationic lipids for oligonucleotide delivery
- Patent Title (中): 用于寡核苷酸递送的基于二酯和三酯的低分子量生物降解阳离子脂质
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Application No.: US14395267Application Date: 2013-04-16
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Publication No.: US09402816B2Publication Date: 2016-08-02
- Inventor: Steven L. Colletti , Matthew G. Stanton
- Applicant: SIRNA THERAPEUTICS, INC.
- Applicant Address: US MA Cambridge
- Assignee: Sima Therapeutics, Inc.
- Current Assignee: Sima Therapeutics, Inc.
- Current Assignee Address: US MA Cambridge
- Agency: Nixon Peabody LLP
- International Application: PCT/US2013/036682 WO 20130416
- International Announcement: WO2013/158579 WO 20131024
- Main IPC: A61K9/51
- IPC: A61K9/51 ; C12N15/113 ; C07C229/28 ; C07C229/30 ; C12N15/11 ; A61K47/48 ; C07C229/12
Abstract:
The instant invention provides for novel cationic lipids of Formula A that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain coupled with inclusion of hydrolysable functionality in the lipid chains to enhance the efficiency and tolerability of in vivo delivery of siRNA.
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