Halting and Reversing the Progression of Facioscapulohumeral Muscular Dystrophy (FSHD)
SLU ID 18-023 | Pyrrolopyridinones
Intellectual Property Status
Seeking
Provisional patent application submitted
Know-how based
Licensee
Development partner
Commercial partner
Investment
University spin out
Background
Facioscapulohumeral Muscular Dystrophy (FSHD) is a rare but devastating disease that affects 1 in 20,000 individuals. Current research indicates that FSHD is caused by the mis-expression of teh DUX4 gene in muscle, which leads to muscle cell death.
Overview
Researchers at Saint Louis University have discovered a series of pyrrolopyridinone compounds that inhibit BET bromodomains. The compounds also repress the expression of DUX4 and its downstream gene targets in FSHD patient-derived muscle cells.
Benefits
The potential benefits of this technology include:
Minimizing the progression of FSHD
Increasing the ability to halt the progression of FSHD.
Increasing the ability to reverse the progrression of FSHD
Increasing quality of life for FSHD patients
Minimizing the symptoms of FSHD
Applications
Potential applications of this technology include developing treatments for Facioscapulohumeral Muscular Dystrophy (FSHD).
Opportunity
Saint Louis University is seeking partners to further develop and commercialize this technology.