The Need:

In the realm of therapeutic development, there's a pressing need for precise targeting of dysregulated ribonucleoprotein (RNP) complexes to combat various diseases, particularly neurodegenerative disorders like ALS, Alzheimer’s, and Huntington’s diseases. Traditional methods often lack specificity and struggle to effectively address the underlying RNA-protein interactions implicated in these conditions.

The Technology:

Our groundbreaking technology, RNA-PROTACs, leverages bPNA-RNA hybrids to orchestrate the degradation of RNA-binding proteins (RBPs) within RNP complexes. By functionalizing bifacial peptide nucleic acids (bPNAs) with E3 ligase ligands and employing triplex hybridization to RNA, our approach enables targeted proteolysis of RBPs docked proximal to specific RNA secondary structure motifs. This innovative strategy capitalizes on endogenous protein degradation pathways, offering a novel therapeutic avenue for diseases driven by dysregulated RNPs.

Commercial Applications:

• Precision targeting of dysregulated RNPs implicated in neurodegenerative disorders.
• Therapeutic intervention for diseases where RNA-protein interactions play a pivotal role.
• Potential application in developing treatments for various cancers and genetic disorders.

Benefits/Advantages:

• Unprecedented precision: Enables selective degradation of RNPs centered at specific RNA secondary structural motifs, minimizing off-target effects.
• Biomimetic approach: Mimics natural mechanisms of RBP proteolysis, enhancing therapeutic efficacy.
• Versatile platform: Can be adapted to target a wide range of disease-associated RNPs, offering flexibility in therapeutic development.
• Enhanced cell penetration: Utilizes bPNA with established cell-penetrating properties, facilitating intracellular delivery and efficacy.
• Potential for novel therapeutic development: Offers a unique approach to addressing diseases with limited treatment options, such as neurodegenerative disorders.