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US 9,066,982 B2Granted
Bacterially-derived, intact minicells that encompass plasmid-free functional nucleic acid for in vivo delivery to mammalian cells
AssigneeEnGeneIC Molecular Delivery Pty Ltd
InventorsHimanshu Brahmbhatt, Jennifer MacDiarmid, Toby Hulf
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Abstract
Intact, bacterially-derived minicells can safely introduce therapeutically effective amount of plasmid-free functional nucleic acid to target mammalian cells. To this end, functional nucleic acid can be packaged into intact minicells directly, without resort to expression constructs, the expression machinery of the host cell, harsh chemicals or electroporation.
Claims16
A composition comprising
- (a) a plurality of intact, bacterially derived minicells, each minicell of said plurality encompassing regulatory RNA that is packaged in said minicell, and
- (b) a pharmaceutically acceptable carrier therefor,
- wherein said regulatory RNA is selected from the group consisting of antisense ssRNA, ribozyme and decoy RNA,
- wherein there is an absence from said minicells of a construct for in situ expression of said regulatory RNA, and
- wherein said plurality contains a therapeutically effective amount of said regulatory RNA.
2 more elements
A method of delivering regulatory RNA, comprising
- (a) providing a plurality of intact, bacterially derived minicells in a pharmaceutically acceptable carrier, each minicell of said plurality encompassing regulatory RNA, and
- (b) bringing minicells of said plurality into contact with mammalian cells such that said mammalian cells engulf minicells of said plurality, whereby said regulatory RNA is released into the cytoplasm of the target cells, wherein said regulatory RNA is selected from the group consisting of antisense ssRNA, ribozyme and decoy RNA, wherein there is an absence from said minicells of a construct for in situ expression of said regulatory RNA, and wherein said plurality contains a therapeutically effective amount of said regulatory RNA.
Dependencies are inferred from the claim text.