Published Technical Disclosure Archive

Targeted Gene Regulation System with Enhanced Cellular Uptake

Publication ID: 24-11857633_0005_PTD
Published: October 28, 2025
Category:Future Evolutions & Paradigm Shifts

Legal Citation

pr1or.art Inc., “Targeted Gene Regulation System with Enhanced Cellular Uptake,” Published Technical Disclosure No. 24-11857633_0005_PTD, Published October 28, 2025, available at https://archive.pr1or.art/24-11857633_0005_PTD
This technical disclosure describes improvements that would be readily apparent to a Person Having Ordinary Skill In The Art (PHOSITA) when considered in combination with the foundational architecture disclosed in U.S. Patent No. 11,857,633.

Summary of the Inventive Concept

A next-generation system for targeted gene regulation, comprising nucleic acid conjugates with microRNAs and phosphorothioate moieties, enabling enhanced cellular uptake and stability for treating diseases and disorders.

Background and Problem Solved

The original patent disclosed phosphorothioate-conjugated miRNAs for treating diseases, but limitations included inefficient cellular uptake and stability. The new inventive concept addresses these limitations by introducing a system with enhanced cellular uptake and stability, enabling more effective targeted gene regulation.

Detailed Description of the Inventive Concept

The system comprises nucleic acid conjugates with microRNAs designed to selectively regulate target genes involved in diseases or disorders. The phosphorothioate moiety enhances cellular uptake and stability of the conjugate, allowing for more efficient regulation. The system can be used for treating diseases, personalized medicine, and identifying target genes for regulation.

Novelty and Inventive Step

The new claims introduce the concept of a system with enhanced cellular uptake and stability, which is not present in the original patent. The use of phosphorothioate moieties to enhance cellular uptake and stability is a non-obvious improvement over the original patent.

Alternative Embodiments and Variations

Alternative embodiments include using different types of nucleic acid conjugates, such as siRNAs or antisense oligonucleotides, or modifying the phosphorothioate moiety to further enhance cellular uptake and stability.

Potential Commercial Applications and Market

The inventive concept has significant commercial potential in the treatment of diseases and disorders, personalized medicine, and gene therapy. Target industries include pharmaceutical, biotechnology, and healthcare.

CPC Classifications

SectionClassGroup
A A61 A61K47/549
A A61 A61K47/548
A A61 A61K48/00
C C12 C12N15/113
C C12 C12N2310/141
C C12 C12N2320/30

Field of Art

Molecular Biology and Pharmaceutical Biotechnology, focusing on nucleic acid therapeutics, gene regulation, and drug delivery systems. Requires advanced degree (PhD/MD) with expertise in molecular biology, biochemistry, and pharmaceutical sciences

Person of Ordinary Skill (PHOSITA) Profile

Expert in nucleic acid design, conjugation strategies, cellular delivery mechanisms, with knowledge of microRNA therapeutics, phosphorothioate chemistry, and drug development techniques

Obviousness Rationale

A PHOSITA would recognize that enhancing cellular uptake and stability of microRNA conjugates through phosphorothioate modifications represents a predictable optimization strategy in nucleic acid therapeutic design. The source patent's foundational work on microRNA conjugates provides a clear technical framework that would naturally motivate exploring chemical modifications to improve cellular penetration and molecular stability. Such incremental improvements are consistent with standard pharmaceutical development approaches in nucleic acid therapeutics.

Obvious Combinations & Variations

Source Patent Element
microRNA conjugates for treating multiple myeloma and inhibiting IL-6 signaling
PTD Variation
Adding phosphorothioate moieties to enhance cellular uptake and conjugate stability
Obviousness Reasoning
Known chemical modification technique to improve nucleic acid therapeutic performance, representing a predictable solution to enhance drug delivery efficiency
Source Patent Element
Specific microRNA sequences like let7a-3p, let7a-5p, miR17-3p
PTD Variation
Creating a library of microRNA conjugates with phosphorothioate modifications for personalized medicine approaches
Obviousness Reasoning
Systematic exploration of conjugation strategies is a standard approach in developing targeted therapeutic platforms, representing an obvious extension of existing research
Source Patent Element
Methods of delivering non-cell penetrating ribonucleic acids
PTD Variation
Introducing phosphorothioate moieties specifically to improve cellular penetration and molecular stability
Obviousness Reasoning
Addressing inherent limitations in cellular delivery is a predictable design challenge with finite known solutions, making chemical modification an obvious optimization strategy
Source Patent Element
microRNA-based gene regulation techniques
PTD Variation
Developing systematic methods for identifying target genes through conjugate design
Obviousness Reasoning
Expanding therapeutic screening methodologies represents a natural progression in translational research, utilizing known molecular biology techniques
Source Patent Element
Nucleic acid conjugation strategies for disease treatment
PTD Variation
Generalizing conjugation approach across different disease contexts with phosphorothioate enhancement
Obviousness Reasoning
Applying consistent chemical modification strategies across multiple disease domains is a standard pharmaceutical development approach, representing an obvious technological extension
35 U.S.C. § 103 Summary: Based on the teachings of US Patent 11857633 and the principles of molecular therapeutics, the claimed variations in nucleic acid conjugate design with phosphorothioate modifications represent obvious extensions of prior art, predictably enhancing cellular uptake and molecular stability through routine chemical engineering techniques known to those skilled in nucleic acid therapeutic development.

Original Patent Information

Patent NumberUS 11,857,633
TitlePhosphorothioate-conjugated miRNAs and methods of using the same
Assignee(s)City of Hope