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US 12,178,855 B2Granted
Compositions and methods for facilitating delivery of synthetic nucleic acids to cells
AssigneeTranslate Bio MA, Inc.
InventorsBalkrishen Bhat, Jia Tay, Saswata Karmakar, Nelson Chau
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Abstract
Provided herein are compositions and methods for facilitating or enhancing delivery of nucleic acids, such as synthetic mRNAs, into cells or tissues. Such compositions and methods may include use of a targeting moiety-conjugated, such as an N-acetylgalactosamine (GalNAc)-conjugated, oligonucleotide to facilitate or enhance delivery.
Claims21
A composition comprising:
- (a) a single-stranded oligonucleotide of 5 to 50 nucleotides in length covalently linked to a targeting moiety;
- and
- (b) a synthetic RNA comprising at least one binding region that is complementary to a contiguous stretch of at least 5 nucleotides of the oligonucleotide, wherein the single-stranded oligonucleotide is non-covalently associated with the synthetic RNA through the at least one binding region in the synthetic RNA;
- and
- wherein the targeting moiety comprises one or more one or more sugar moieties and has of one of the following formulae:
- wherein:
- each instance of LA, LB, and LC is independently either absent or is a linker selected from the group consisting of substituted alkylene, substituted alkenylene, substituted alkynylene, substituted heteroalkylene, substituted heteroalkenylene, substituted heteroalkynylene, substituted acylene, substituted carbocyclylene, substituted heterocyclylene, substituted arylene, substituted heteroarylene, and combinations thereof;
- and
- each instance of G1, G2, and G3 is independently a ligand.
6 more elements
A pharmaceutical preparation comprising a complex, wherein the complex comprises:
- a single-stranded nucleic acid non-covalently associated with an oligonucleotide;
- wherein the single-stranded nucleic acid is at least 50 to 10,000 nucleotides longer than the oligonucleotide;
- and
- wherein the oligonucleotide is covalently linked to one or more targeting moieties, wherein the one or more targeting moieties is of one of the following formulae:
- wherein:
- each instance of LA, LB, and LC is independently either absent or is a linker selected from the group consisting of substituted alkylene, substituted alkenylene, substituted alkynylene, substituted heteroalkylene, substituted heteroalkenylene, substituted heteroalkynylene, substituted acylene, substituted carbocyclylene, substituted heterocyclylene, substituted arylene, substituted heteroarylene, and combinations thereof;
- and
- each instance of G1, G2, and G3 is independently a ligand.
5 more elements
Dependencies are inferred from the claim text.