Olfactory delivery scaffolds using antisense oligonucleotides and methods for making and using same
Exemplary olfactory delivery scaffolds may include 1) an olfactory targeting component, stimulant, or odorant that is recognized by the olfactory nerves via, for example, a smell response, 2) a molecule with biological activity, a therapeutic component, and/or drug (sometimes collectively referred to herein as a “therapeutic component”), and 3) a linker component that links the olfactory targeting component and therapeutic component together. The olfactory delivery scaffolds may be used to deliver a molecule with biological activity and/or a therapeutic component to a subject's neurological system through the olfactory pathway and pharmaceutical compositions useful in the treatment of neurological and/or neurodegenerative diseases.
1 . An olfactory delivery scaffold compound which provides an olfactory targeting component, a linker and a therapeutic component, wherein the olfactory delivery scaffold compound is a compound of Formula (G)
wherein R is selected from the group consisting of
wherein the * symbol marks a carbon atom that attaches by a bond to the remaining compound,
wherein X is O or NH,
wherein ASO is an antisense oligonucleotide attached at the 3′ or 5′ position.
2 . The compound of claim 1 , wherein R is
3 . The compound of claim 1 , wherein R is
4 . The compound of claim 1 , wherein R is
5 . The compound of claim 1 , wherein R is