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 addressing disease-associated alternative splicing patterns, necessitating the development of new technologies that can target and regulate splicing processes.
Innovation Solution
Development of compounds that modulate nucleic acid splicing by binding to pre-mRNA or components of the splicing machinery, such as snRNPs, to alter splicing events and regulate gene product levels, which can be used to prevent or treat various diseases including cancers and neurological disorders.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Adaptability or versatility
If oligonucleotide targeting or gene therapy is used to modulate RNA expression, then RNA expression can be regulated, but the ability to effectively address disease-associated alternative splicing patterns is limited
Solution Approach 1:
The patent employs small molecule compounds as intermediaries that bind to splicing factors (such as hnRNPs and SR proteins) to modulate alternative splicing. These small molecules act as mediators between the therapeutic goal and the splicing machinery, providing a new mechanism to address alternative splicing patterns that oligonucleotide therapies cannot effectively target.
2Adaptability or versatility
If small molecule compounds are developed to target splicing, then new therapeutic capabilities are achieved, but the complexity of the splicing machinery makes effective targeting difficult
Solution Approach 1:
The patent focuses on targeting specific functional domains or interaction interfaces within splicing factors using small molecule compounds. Rather than attempting to control the entire splicing machinery, the approach applies local quality by designing molecules that specifically bind to particular regions of splicing proteins, thereby modulating splicing events with greater precision despite the overall complexity of the system.
Data Source
AI summary
The present disclosure features compounds (I) and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof.