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US 2026/0201363 A1Published application
Compositions containing oligonucleotides with theranostic applications
AssigneeSixfold Bioscience Ltd.
InventorsSalesia WERNER, James Luke RUSHWORTH, Ioanna MYLONAKI, Auréle LACROIX, Piotr KLIMKOWSKI, George William FOOT, Anna Perdrix ROSELL
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Abstract
The disclosure provides therapeutic compositions that include theranostic nucleic acid nanostructures for delivery of CA cargo and methods of using the same.
Claims167
A composition comprising:
- a nucleic acid analyte further comprising a nucleic acid barcode, wherein said nucleic acid barcode allows for the detection and unambiguous identification of the nucleic acid analyte in a biological sample, and wherein said nucleic acid barcode is not encapsulated in a lipid nanoparticle formulation.
A composition comprising:
- (i) a nucleic acid nanostructure self-assembled from two or more oligonucleotide strands that are not nucleic acid therapies (NATs), said nanostructure not encapsulated in the lipid nanoparticle formulation;
- (ii) a cargo molecule that is linked to said nucleic acid nanostructure;
- and
- (iii) optionally a nucleic acid barcode that is part of or linked to said nucleic acid nanostructure.
1 more element
A composition comprising an oligonucleotide of formula (I), or a salt thereof:
- wherein:
- F is an optional 5′ terminal cargo that is either absent or present;
- T is an optional 3′ terminal cargo that is either absent or present;
- z is 1 or any positive integer greater than 1;
- and p are each independently, and independently for each occurrence, 0 or any positive integer greater than 0;
- m, g, h, and n are each independently, and independently for each occurrence, 0 or 1;
- for any o>l, g≤m for each but the first occurrence;
- for any p>l, h≤n for each but the first occurrence;
- S1 and S2 are nucleotide sequences, each independently comprising at least 1 and not more than 100,000 nucleotides in length, where for any o>1 or p>1 or z>1 or combinations thereof:
- (i) each occurrence of S1, independently, represents a nucleic acid sequence identical to or different to the nucleic acid sequence of the first occurrence of S1;
- (ii) each occurrence of S2, independently, represents a nucleic acid sequence identical to or different to the nucleic acid sequence of the first occurrence of S2;
- r is a covalent or non-covalent linkage, where for any o>1 or p>1 or z>1 or combinations thereof each occurrence of r represents a linkage identical to or different to the linkage of the first occurrence of r;
- and
- B is a nucleic acid barcode comprising a nucleic acid sequence, where for z>1 each occurrence of B, independently, comprises a nucleic acid sequence identical to or different to the nucleic acid sequence of the first occurrence of B;
- and
- wherein said oligonucleotide of formula (I) is not encapsulated in a lipid nanoparticle formulation.
13 more elements
A composition comprising:
- an alkyl phosphonamidite of formula (II):
- wherein CxHY denotes an alkyl chain with the general formula CnH2n−1, where x can be anywhere between 1 and 20 and x can be anywhere between 3 and 39.
A composition comprising:
- an alkyl phosphonamidite of formula (III):
- wherein R is a nucleotide base and CxHY denotes an alkyl chain with the general formula CnH2n−1, where x can be anywhere between 1 and 20 and x can be anywhere between 3 and 39.
A composition comprising:
- a phosphitylating reagent of formula (IV):
- wherein R is a moiety that can modulate the physicochemical properties of an oligonucleotide. This includes moieties selected from the group consisting of O-alkylamino, O-alkylalkoxy, protected O-alkylamino, O-alkylaminoalkyl, O-alkyl imidazole, O-alkyl guanidine, polyamines of the formula (O-alkyl)m, where m is about 1 to 20 and polyethers of the formula (O-alkyl)m, where m is about 1 to 20. Preferred ethers are polyethylene glycols (PEGs).
A composition comprising:
- a nucleic acid nanostructure not formulated in a lipid nanoparticle comprising or linked to a nucleic acid barcode, and
- optionally, a cargo molecule that is covalently linked to the nucleic acid nanostructure.
A composition comprising:
- a nucleic acid nanostructure, and
- a layer of hydrophilic molecules that are electrostatically bound to the nucleic acid phosphate backbone.
A composition comprising
- a nucleic acid nanostructure, and
- a layer of molecules that can mediate endosomal escape, which are electrostatically bound to the nucleic acid phosphate backbone.
A composition comprising
- a nucleic acid nanostructure, and
- a layer of primary amine-containing small molecules that have a therapeutic effect.
Dependencies are inferred from the claim text.