Compounds and methods for trans-membrane delivery of molecules
AssigneeAposense Ltd.
InventorIlan Ziv
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Abstract
A novel delivery system for drugs, and especially macromolecules such as proteins or oligonucleotides through biological membranes is provided, and specifically delivery of siRNA. The delivery system comprises conjugation of the macromolecule drug to a moiety that enables effective passage through the membranes. Respectively, novel compounds and pharmaceutical compositions are provided, utilizing said delivery system. In one aspect of the invention, the compounds may be utilized in medical practice, for example, in delivery of siRNA or antisense oligonucleotides across biological membranes for the treatment of medical disorders.
Claims21
A conjugate according to general Formula (I),
- or a pharmaceutically acceptable salt, hydrate, or solvate thereof, or a solvate or hydrate of the salt, wherein:
- D is a drug selected from a group consisting of a small-molecule drug, a peptide, a protein, a native or modified, single-stranded or double-stranded DNA or RNA, siRNA, and antisense oligonucleotide (ASO);
- y, z and w are each an integer, independently selected from 0, 1, 2, 3, 4, 5, or 6, wherein at least one of y, z or w is different from 0;
- E, E′ and E″ are the same or different, and are defined by the Formula (VII):
- U is —O—;
- Q1 and Q2 are each independently selected from null, ester, amide, carbamate, urea, disulfide, or amine;
- L1, L2, and L3 are each independently selected from null, a linear C1-14 alkyl or heteroalkyl, a cyclic or branched C3-14 alkyl or heteroalkyl, C5-6 aryl or heteroaryl, or —(O—CH2—CH2)u;
- wherein u is an integer of 1, 2, 3, 4, or 5;
- wherein at least one of Q1, Q2, L1, L2, and L3 is not null;
- R and R′ are each hydrogen;
- W is —O—;
- G is null, a secondary or tertiary amine groups;
- d and k are each an integer, independently selected from 0, 1, 2, 3, 4, 5, or 6.
Dependencies are inferred from the claim text.