New York, NY – June 7, 2006 – The Spinal Muscular Atrophy Foundation today announced that they have initiated a research and development collaboration with PTC Therapeutics, Inc. (PTC). The collaboration is designed to leverage PTC’s proprietary Gene Expression Modulation by Smallmolecules (GEMS) technology to identify and develop new small molecule therapeutics for use in the treatment or prevention of spinal muscular atrophy (SMA), a neuromuscular disease and the leading genetic cause of death among infants and toddlers. 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. The application of these compounds and technology in SMA follows on the work of Christian Lorson, PhD, Assistant Professor of Veterinary Pathobiology at the University of Missouri, who observed that certain compounds could improve the stability and distribution of the abnormal SMN2 protein in vitro, and proposed that such changes could be harnessed to modulate disease severity in SMA.
About Spinal Muscular Atrophy
Spinal muscular atrophy is a genetic, motor neuron disease characterized by the wasting of skeletal muscles. Caused by progressive degeneration of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Spinal muscular atrophy is often compared to polio because the same spinal cord cells are attacked in each disease. Over time, its victims continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe.
About the Spinal Muscular Atrophy Foundation
Founded in 2003, the Spinal Muscular Atrophy Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment and cure for spinal muscular atrophy through targeted funding of clinical research and novel drug development efforts. Since its inception, the Foundation has awarded over $30 million in sponsored research agreements. In addition, the Foundation is committed to raising awareness and generating support for increased research efforts in SMA among the leaders of industry and government. For more information on the Spinal Muscular Atrophy Foundation, visit www.smafoundation.org or call (646) 253-7100.
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes. Posttranscriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities. PTC’s current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.
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