Small Molecules to Facilitate Therapeutic Exon Skipping

Text republished from the UCLA Office of Intellectual Property & Industry Sponsored Research: https://techtransfer.universityofcalifornia.edu/NCD/20445.html

A number of antisense oligonucleotide agents are currently in clinical trials for a wide range of diseases. Antisense technology is broadly used by the pharmaceutical industry as a tool for functional genomics and for highly specific drugs in different therapeutic areas. Antisense oligonucleotides in clinical trials are frequently found to be too inefficient to cause a sufficient amount of exon skipping to be therapeutically effective. To date, no molecule that can increase the efficiency of antisense mediated skipping has been identified.

Researchers at UCLA have discovered a series of compounds that facilitate therapeutic exon skipping. The compounds were derived from FDA approved libraries or known biologically active molecule libraries. The molecules were identified via a small molecule library screen using a cell reporter assay. Some compounds have been demonstrated to increase the amount of mRNA that is skipped in the presence of antisense therapeutics.

APPLICATIONS

• Enhancement of the therapeutic effect of antisense oligonucleotides when used as a combination treatment
  
• Increase in the amount of mRNA that is skipped in the presence of antisense therapeutics
  

ADVANTAGES

• Enhancement of the therapeutic effect of antisense treatments that are currently too inefficient to be effective
  
• The compounds were derived from FDA-approved libraries of known biologically active molecule libraries
  

Image credit: RNA Molecule, Blausen.com staff. "Blausen gallery 2014". Wikiversity Journal of Medicine. DOI:10.15347/wjm/2014.010. ISSN 20018762