Investigators at UCLA have discovered a series of small molecules to facilitate therapeutic exon skipping. The use of these identified molecules may enhance the effectiveness of antisense oligonucleotide agents currently in clinical development.
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.
The compounds have been identified from a small molecule library screen using a cell reporter assay. Some compounds have undergone further testing in cell culture and are able to increase the amount of mRNA that is skipped in the presence of antisense entities.
|United States Of America||Published Application||20160220538||08/04/2016||2009-381|
Additional Patent Pending