Olfactory delivery scaffolds using antisense oligonucleotides and methods for making and using same
Inventors
Cooper, Kelvin • LAK, Parnian • Renault, Renaud
Assignees
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Publication Number
US-12629423-B2
Publication Date
2026-05-19
Expiration Date
Abstract
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.
Core Innovation
The invention relates to an olfactory delivery scaffold system for delivering therapeutic agents to the central nervous system via the olfactory pathway without crossing the blood-brain barrier. The scaffold is presented as an olfactory targeting component, a therapeutic component, and an enzyme-cleavable linker that releases separate targeting and therapeutic fragments in nasal mucus and/or the olfactory system.
The olfactory delivery scaffold compound is defined by Formula (G) and provides an olfactory targeting component, a linker, and a therapeutic component. X is O or NH, and the therapeutic component includes an antisense oligonucleotide (ASO) attached at the 3′ or 5′ position.
The description further includes multiple cleavage schemes, including single-component prodrug-like scaffolds cleaved by esterase, amidase, nuclease, hydrolase, and cathepsin, and also describes pH-sensitive linkers. The framework further specifies substituent R through depicted structures and related sub-formulas, including embodiments with biological or therapeutic moieties and an olfactory-receptor interacting moiety.
Claims Coverage
The provided claims center on an olfactory delivery scaffold compound defined by Formula (G) with an olfactory targeting component, a linker, and a therapeutic component. The independent claim coverage emphasizes three inventive features: the Formula (G) scaffold, the restriction that X is O or NH, and ASO attachment at the 3′ or 5′ position.
Formula (G) olfactory delivery scaffold
An olfactory delivery scaffold compound providing an olfactory targeting component, a linker and a therapeutic component, wherein the compound is Formula (G).
Linker heteroatom X is O or NH
X is O or NH as part of the Formula (G) definition.
Antisense oligonucleotide attachment at 3′ or 5′ position
ASO is an antisense oligonucleotide attached at the 3′ or 5′ position.
The claim coverage is anchored on a Formula (G) olfactory delivery scaffold with an olfactory targeting component, a linker, and a therapeutic component, with X restricted to O or NH and ASO conjugated at the 3′ or 5′ position.
Stated Advantages
Deliver therapeutic agents to the central nervous system via the olfactory pathway without crossing the blood-brain barrier.
Documented Applications
Olfactory delivery of therapeutic agents to the central nervous system/brain using the olfactory pathway, including release in nasal mucus and/or the olfactory system.
Alzheimer’s-related therapeutic targeting, including amyloid-β targeting agents.
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