Treating Facioscapulohumeral Muscular Dystrophy
SLU ID 13-019 | Small molecule inhibition of BET bromodomain proteins
Intellectual Property Status
Seeking
Patented
Know-how based
Licensee
Development partner
Commercial partner
Investment
University spin out
Background
Facioscapulohumeral Muscular Dystrophy (FSHD) is characterized by epigenetic changes resulting in the aberrant expression of the DUX4 gene in muscle. Suppression of DUX4 expression is considered a therapeutic target for FSHD. However, the mechanisms responsible for DUX expression are poorly understood and few targets amenable to small molecule drug intervention have been described.
Overview
Researchers at Saint Louis University have identified inhibitors of BET bromodomain proteins that block induction of DUX4 expression and activity.
Benefits
The potential benefits of this technology include:
Increasing the potential treatments for FSHD
Minimizing muscle degeneration in FSHD patients
Applications
The potential applications of this technology include:
Developing treatments for Facioscapulohumeral Muscular Dystrophy (FSHD)
Opportunity
Saint Louis University is seeking a partner to further develop and commercialize this technology.