Back to results
US 2022/0090066 A1Published application
Cell penetrating molecule
OwnerMatthew Wood, Graham McClorey +4inventors
InventorsMatthew Wood, Graham McClorey, Michael Gait, Peter Jarver, Amer Salah, Fazel Shabanpoor
Life of this patent
Estimating the term…
Abstract
The present invention relates to cell penetrating molecules comprising two oligonucleotide cargo molecules.
Claims19
A molecule having the structure of formula (I):
- (CPP)-(S1)-(L1)-(S2)-(L2)
- or formula (II)
- (L1)-(S1)-(CPP)-(S2)-(L2);
- wherein:
- (a) (CPP) is a cell penetrating peptide arranged in the N to C terminal direction;
- (S1) and (S2) are spacer moieties which are independently present or absent;
- (L1) is a first linking moiety;
- (L2) is a second linking moiety;
- (b) each of (L1) and (L2) is independently attached to a separate cargo molecule which comprises an oligonucleotide;
- (c) each said oligonucleotide comprises a sequence which specifically hybridises to a target sequence and either:
- (i) the oligonucleotide attached to (L1) is identical to the oligonucleotide attached to (L2);
- or
- (ii) the oligonucleotide attached to (L1) and the oligonucleotide attached to (L2) each comprise a sequence which specifically hybridises to the target sequence;
- or
- (iii) the oligonucleotide attached to (L1) comprises a sequence which specifically hybridises to the target sequence in an RNA molecule and the oligonucleotide attached to (L2) comprises a sequence which specifically hybridises to a second, different target sequence in the same RNA molecule;
- or
- (iv) the oligonucleotide attached to (L1) comprises a sequence which specifically hybridises to the target sequence in an RNA molecule, the sequence of which RNA molecule is encoded by a sequence in the genome of a cell which at least partially overlaps on the same or the opposite DNA strand with a sequence in said genome which encodes a second RNA molecule, and the oligonucleotide attached to (L2) comprises a sequence which specifically hybridises to a second target sequence in said second RNA molecule.
14 more elements
Dependencies are inferred from the claim text.