BMP-Encoding RNA for Bone Regeneration

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Solution Overview

Problem

Current methods for treating bone diseases and injuries are limited by the need for expensive recombinant proteins and the risk of side effects, as well as challenges with gene delivery such as immunogenicity and low gene transfer efficiency.

Innovation Solution

A pharmaceutical composition comprising a polyribonucleotide (RNA) that encodes a bone morphogenetic protein (BMP), specifically BMP-2 or BMP-7, is administered to treat or prevent bone diseases, disorders, or injuries, and to induce osteogenic differentiation and bone regeneration.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If recombinant BMP proteins are used to treat bone diseases, then bone regeneration is achieved, but treatment cost increases and side effects occur

Engineering Contradiction:
Improvebone regeneration efficacyVSAvoidside effects and cost
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent uses RNA copies of the BMP gene to instruct cells to produce BMP proteins endogenously, rather than administering recombinant BMP proteins directly. This RNA-based approach replicates the natural gene expression process, allowing the body to produce its own growth factors, thereby avoiding the side effects and high costs associated with recombinant protein therapy while maintaining bone regeneration efficacy

Inventive Principle:
Principle #26Copying

Solution Approach 2:

The patent introduces RNA as an intermediary molecule that bridges the gap between genetic information and protein production. Instead of directly administering BMP proteins or integrating DNA into the genome, the RNA serves as a temporary mediator that translates the BMP gene sequence into functional proteins in the cytoplasm, achieving bone regeneration without the drawbacks of direct protein administration or viral gene therapy

Inventive Principle:
Principle #24Intermediary (Mediator)

2Duration of action of moving object

If DNA-based gene therapy is used to deliver BMP genes, then sustained protein production is achieved, but immunogenicity and low gene transfer efficiency occur

Engineering Contradiction:
Improvesustained protein productionVSAvoidgene transfer efficiency and immunogenicity
Core Design Contradiction:
Duration of action of moving objectVSReliability

Solution Approach 1:

Instead of using DNA as the starting material for gene therapy (which requires nuclear entry and transcription), the patent inverts the approach by directly introducing RNA into the cytoplasm. This bypasses the problematic DNA delivery step and nuclear import requirements, achieving sustained protein production through direct translation of the introduced RNA while avoiding the immunogenicity and low efficiency associated with DNA-based gene therapy

Inventive Principle:
Principle #13The other way round (Inversion)

Solution Approach 2:

The patent extracts the essential functional element (the BMP coding sequence) from the DNA context and delivers it directly as RNA to the cytoplasm. This extraction approach removes the problematic aspects of DNA delivery (nuclear entry, transcriptional regulation, genomic integration) while retaining the ability to produce sustained BMP protein expression through direct translation of the introduced RNA

Inventive Principle:
Principle #2Taking out (Extraction)

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The use of BMP-encoding RNA, particularly in its chemically modified form, achieves effective osteogenesis and bone regeneration, allowing for a single treatment to achieve sound treatment or prevention of bone-related conditions without the limitations of DNA-based gene therapy.

Implementation Method 1

A pharmaceutical composition comprising a polyribonucleotide (RNA) that encodes a bone morphogenetic protein (BMP), specifically BMP-2 or BMP-7, is administered to treat or prevent bone diseases, disorders, or injuries, and to induce osteogenic differentiation and bone regeneration

Methodology Applied
Scientific EffectGene expression:

Data Source

PatentUS12263229B2Induction of osteogenesis by delivering BMP encoding RNA
Publication Date: 2025.04.01 ETHRIS
  • US12263229B2 patent drawing
  • US12263229B2 patent drawing
  • US12263229B2 patent drawing

AI summary

The present invention relates to a pharmaceutical composition comprising a polyribonucleotide (RNA) with a sequence which encodes a bone morphogenetic protein (BMP) for use in (i) treating or preventing a bone disease, bone disorder or bone injury; and/or (ii) inducing or enhancing osteogenic differentiation, osteogenesis, ossification, bone regeneration and/or bone morphogenesis in a patient. The present invention also relates to the respective BMP encoding RNAs (BMP RNAs), in particular in its chemically modified form. The present invention also relates to complexes which comprise or are complexed with the BMP RNA, in particular to the respective transfection complexes like lipofection, magnetofection and magnetolipofection complexes. The present invention further relates to a carrier and carrier body to which the RNA or complex has been loaded and to a pharmaceutical composition comprising said carrier or carrier body. The present invention further relates to a matrix or scaffold for sustained mRNA delivery and its application in bone regeneration in vivo, ex vivo and in vitro.