Small Molecules to Facilitate Therapeutic Exon Skipping
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
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
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.
Inventor: NELSON, Stanley F. | MICELI, Carrie | MORAN, Miriana
Priority Number: WO2013033407A9
IPC Current: A61K004800 | A61K0031415 | A61K00314166 | A61K00317088 | A61K00317105
Assignee Applicant: The Regents of the University of California
Title: IDENTIFICATION OF SMALL MOLECULES THAT ENHANCE THERAPEUTIC EXON SKIPPING | IDENTIFICATION DE PETITES MOLÉCULES QUI AMÉLIORENT LE SAUT D'EXON THÉRAPEUTIQUE
Usefulness: IDENTIFICATION OF SMALL MOLECULES THAT ENHANCE THERAPEUTIC EXON SKIPPING | IDENTIFICATION DE PETITES MOLÉCULES QUI AMÉLIORENT LE SAUT D'EXON THÉRAPEUTIQUE
Summary: The method is useful for enhancing exon skipping in an mRNA of interest; and treating a subject that has Duchenne muscular dystrophy (DMD), or is a nonhuman model of DMD, where the subject is human (claimed).
Disease Diagnostic/Treatment
Other Disease
20160220538
State of Development 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. Background 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. Tech ID/UC Case 20445/2009-381-0 Related Cases 2009-381-0
USA
