PTC, Roche Team Up Against SMA

Swiss biotechnology company Roche, and PTC Therapeutics in South Plainfield, N.J., on Nov. 29, 2011, announced a collaboration through which the two companies will work together to advance drug development for spinal muscular atrophy(SMA).

Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC’s SMA program. PTC currently has three small-molecule compounds in preclinical development, as well as backup compounds.

“Having been a partner with Roche for several years, we have every confidence that the combination of our own expertise and Roche’s considerable capabilities in clinical development, biomarkers and diagnostics will help us maximize the potential for this program,” said Stuart W. Peltz, president and CEO of PTC Therapeutics, in a PTC press release.

PTC’s compounds increase levels of needed protein

SMA is a genetic neuromuscular disease that causes muscle-controlling nerve cells (motor neurons) in the spinal cord to die, resulting in weakness and eventual paralysis of voluntary muscles, including those involved in breathing.

The molecular basis of the disease is a flawed or missing SMN1 gene, which results in insufficient amounts of SMN protein. (SMN stands for "survival of motor neurons.") It’s thought that increasing levels of the SMN protein will provide therapeutic benefit in SMA.

The compounds currently under development in PTC’s SMA program treat the underlying cause of the disorder. Early testing in research models of the disease has shown that the compounds increase SMN levels in the central nervous system, muscles and other tissues.

Meaning for people with SMA

The Roche-PTC Therapeutics collaboration is expected to provide a boost to PTC’s small-molecule experimental therapies for SMA, with the potential to advance one or more compounds to clinical trials.

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