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Publication Number
US-12214043-B2
Publication Date
2025-02-04
Expiration Date
Abstract
Disclosed herein is a delivery vehicle based on DNA-inspired Janus based nanotubes (JBNTs) for anti-viral treatment. The nanoparticles (NPs) are based the JBNTs conjugated with targeting moieties such as small molecules, aptamers, and peptides.
Core Innovation
The invention relates to DNA-inspired Janus-based nanotubes (JBNTs) and JBNPs as antiviral delivery vehicles for short interfering RNA (siRNA), configured to achieve endosomal escape and inhibition of viral gene expression. The disclosure includes self-assembled Janus base nanotubes comprising compounds of Formulas (I)–(VIII) and related amino acid and β-amino acid analogs, including D- and L-isomers, with various protecting groups, salts, and conservative substitutions.
The disclosed compositions provide a therapeutic agent X conjugated to a DNA-inspired Janus-based nanotube nanoparticle, including embodiments in which X is folic acid. The platform further includes linker options, including L being absent, and linker groups that may be acid-cleavable, reducible, or stimuli linkers, together with targeting moieties such as surface targeting modifiers and organelle-specific targeting molecules.
The document additionally describes JBNT-containing compositions having a specified pH range. It states that therapeutic agent loading and release and intracellular delivery behavior can be supported by the nanotube platform, and it connects delivery performance to mechanisms such as endosomal escape, including a proton sponge effect.
Claims Coverage
The provided independent claims center on 2 inventive features: a Formula (I) composition with L absent and X defined as folic acid, and a dependent refinement that targets a molecule using enumerated targeting-molecule classes.
Formula (I) composition with folic acid and L absent
A composition comprising a compound of Formula (I) or a pharmaceutically acceptable salt thereof, wherein R1 is H or CH3, R2 is (CH2)j, (CH2CH2O)k or (CH2CH2NH)m, j, k, and m independently represent 0–200 of repeating moieties, L is absent, X is folic acid, and the composition has a pH of about 1 to about 10.
Targeted therapeutic agent directed to particular cellular compartments or organelles
The composition further wherein the therapeutic agent targets a molecule using one of: a surface targeting modifier, a membrane dipeptidase targeting molecule, an endoplasmic reticulum targeting molecule, a mitochondrial membrane targeting molecule, or a nucleus targeting molecule.
Overall, the claim coverage centers on a JBNT-related composition defined by a specific Formula (I) substituent scheme with linker L absent, using folic acid as the therapeutic agent X, and setting a composition pH of about 1 to about 10, with dependent claim coverage further restricting the therapeutic agent to targeted delivery using enumerated surface and organellar targeting molecule classes.
Stated Advantages
Low cytotoxicity and low immunogenicity.
Improved or efficient endosomal escape associated with a proton sponge effect.
Active targeting via targeting moieties.
Controlled release supported by the disclosed linker and platform concepts.
Inhibition of viral gene expression.
Tissue pre-delivery for protection.
Significantly lower cytotoxicity versus conventional vehicles.
Documented Applications
Antiviral therapy proof-of-concept, including reference to COVID-19.
Methods of treating viral infections, including COVID-19.
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