Enhanced Phosphorothioate-conjugated miRNAs for Improved Disease Treatment
Legal Citation
Summary of the Inventive Concept
The inventive concept enhances phosphorothioate-conjugated miRNAs for treating multiple myeloma and inhibiting IL-6 signaling by introducing improved stability, potency, and bioavailability through modified phosphorothioate linkers.
Background and Problem Solved
The original patent disclosed phosphorothioate-conjugated miRNAs for disease treatment, but limitations included reduced cellular uptake, off-target effects, and varying potency. The new inventive concept addresses these limitations by incorporating optimized phosphorothioate linkers, resulting in improved treatment outcomes.
Detailed Description of the Inventive Concept
The enhanced phosphorothioate-conjugated miRNAs comprise a microRNA selected from let7a-3p, let7a-5p, miR17-3p, miR17-5p, and miR218-5p, and a phosphorothioate linker modified to improve stability, potency, and bioavailability. The linker modifications enable increased cellular uptake, reduced off-target effects, and enhanced therapeutic efficacy. The inventive concept can be applied to various disease treatments, including multiple myeloma and IL-6 signaling inhibition.
Novelty and Inventive Step
The new claims introduce novel phosphorothioate linker modifications, which provide improved stability, potency, and bioavailability compared to the original patent. These modifications constitute an inventive step, as they address specific limitations of the original patent and provide a significant improvement in treatment outcomes.
Alternative Embodiments and Variations
Alternative embodiments may include varying the microRNA selection, linker modification strategies, or conjugation methods to further optimize the inventive concept. Variations could also involve combining the enhanced phosphorothioate-conjugated miRNAs with other therapeutic agents or delivery systems.
Potential Commercial Applications and Market
The enhanced phosphorothioate-conjugated miRNAs have significant commercial potential in the treatment of multiple myeloma and other diseases, with potential applications in the biotechnology and pharmaceutical industries. The target market includes hospitals, research institutions, and biotech companies focused on disease treatment and gene therapy.
CPC Classifications
| Section | Class | Group |
|---|---|---|
| A | A61 | A61K47/549 |
| A | A61 | A61K47/548 |
| A | A61 | A61K48/00 |
| C | C12 | C12N15/113 |
| C | C12 | C12N2310/141 |
| C | C12 | C12N2320/30 |
Section 103 Obviousness Analysis (PHOSITA)
Field of Art
Molecular Biology and Therapeutic RNA Delivery, specifically focusing on microRNA conjugation technologies for cellular therapeutics, with expertise in nucleic acid modification, drug delivery systems, and targeted molecular interventions
Person of Ordinary Skill (PHOSITA) Profile
A researcher or biotechnology professional with advanced degrees in molecular biology, biochemistry, or pharmaceutical sciences, possessing expertise in RNA modification techniques, conjugation strategies, and understanding of cellular delivery mechanisms
Obviousness Rationale
A person having ordinary skill would recognize that the PTD's phosphorothioate linker modifications represent predictable engineering variations to enhance miRNA delivery and stability. The source patent establishes the foundational miRNA conjugation approach, and the PTD merely extends this concept through routine optimization of linker chemistry. These modifications would be considered obvious improvements within the standard problem-solving toolkit of molecular biology researchers seeking to enhance therapeutic RNA delivery.
Obvious Combinations & Variations
Original Patent Information
| Patent Number | US 11,857,633 |
|---|---|
| Title | Phosphorothioate-conjugated miRNAs and methods of using the same |
| Assignee(s) | City of Hope |