This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
COMPOSITIONS AND METHODS FOR DELIVERY OF THERAPEUTIC AGENTS
申请人:ModernaTX, Inc.
公开号:EP3386484A1
公开(公告)日:2018-10-17
GENE TRANSFER AGENT COMPOSITION CONTAINING POLYAMIDOAMINE DENDRON
申请人:KONO Kenji
公开号:US20120053233A1
公开(公告)日:2012-03-01
The present invention provides a gene transfer agent, a gene transfer kit, and a gene transfer method excellent in safety and transfer efficiency. Specifically, the present invention provides a gene transfer agent composition, the gene transfer agent composition including a compound represented by any one of the following formulae DL-G1 to DL-G4: DL-G1: R
1
R
2
NX(XH
2
)
2
; DL-G2: R
1
R
2
NX(X(XH
2
)
2
)
2
; DL-G3: R
1
R
2
NX(X(X(XH
2
)
2
)
2
)
2
; and DL-G4: R
1
R
2
NX(X(X(X(XH
2
)
2
)
2
)
2
)
2
(X represents —CH
2
CH
2
CONHCH
2
CH
2
N—), in which: R
1
represents an unsaturated long-chain aliphatic group; and R
2
represents an unsaturated long-chain aliphatic group or a saturated long-chain aliphatic group in the formulae.
COMPOSITIONS AND METHODS FOR DELIVERY OF AGENTS
申请人:ModernaTX, Inc.
公开号:US20180028664A1
公开(公告)日:2018-02-01
This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.