Small Molecule Compounds Modulating Pre-mRNA Splicing
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Solution Overview
Problem
Current therapies for modulating RNA expression, such as oligonucleotide targeting and gene therapy, face challenges in effectively regulating alternative splicing patterns associated with diseases, necessitating the development of new technologies that can target splicing processes.
Innovation Solution
The development of specific compounds, represented by Formulas (I), (II), and (III), which can modulate nucleic acid splicing by binding to pre-mRNA or components of the splicing machinery, thereby altering splicing events and regulating gene product levels.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If oligonucleotide targeting or gene therapy is used to modulate RNA expression, then RNA expression can be regulated, but the therapies face challenges in effectively regulating alternative splicing patterns associated with diseases
Solution Approach 1:
The patent changes the fundamental parameter of the therapeutic agent from large molecules (oligonucleotides, gene therapy vectors) to small organic compounds. This parameter change enables the compounds to penetrate cell membranes and interact with splicing machinery in ways that larger molecules cannot, directly addressing the effectiveness challenge while simplifying the delivery mechanism.
Solution Approach 2:
The small molecule compounds serve as intermediary agents that bind to splicing factors or pre-mRNA, facilitating or inhibiting splicing events. These compounds act as mediators between the therapeutic goal and the splicing machinery, providing a more effective and simpler mechanism than direct oligonucleotide targeting.
2Adaptability or versatility
If current therapy modalities are used, then RNA expression modulation is achieved, but new technologies are needed to effectively target splicing processes
Solution Approach 1:
The patent segments the splicing process into specific binding sites and interaction points that can be targeted by small molecules. By dividing the complex splicing machinery into discrete targetable elements, the invention enables selective modulation of splicing events without requiring complex therapeutic systems.
Solution Approach 2:
The small molecule compounds exhibit multi-functionality, capable of binding to different splicing factors and pre-mRNA sequences with high affinity. This universal binding capability allows a single compound class to target multiple splicing events, providing versatility without requiring multiple specialized therapies.
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
These compounds effectively modulate splicing processes, allowing for the prevention and treatment of various diseases, including proliferative, neurological, and metabolic disorders, by specifically targeting and altering splicing events, thereby regulating gene expression.
Implementation Method 1
binding to pre-mRNA or components of the splicing machinery
Data Source
AI summary
The present disclosure features compounds and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof.