The collaboration is designed to leverage PTC’s proprietary Gene Expression Modulation by Small molecules (GEMS) technology to identify and develop small molecule therapeutics for use in the treatment or prevention of spinal muscular atrophy (SMA).
Under the terms of the agreement, the SMA Foundation will provide up to $1.6 million in funding to PTC based upon completion of certain milestones.
"PTC has demonstrated a strong commitment to SMA and other pediatric orphan diseases," said Loren Eng, President of the SMA Foundation.
"We believe PTC’s scientific assets and proven accomplishments make them an ideal partner in our efforts to identify treatments for SMA."
PTC’s GEMS technology exploits the regulatory mechanisms found in the untranslated regions of messenger RNA for the identification of small-molecule drugs that can treat diseases by selectively increasing or decreasing the expression of key proteins by modulating post-transcriptional mechanisms.
Another SMA Foundation research project will explore the application of PTC’s nonsense suppression compounds and technology to SMA.