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12:00 AM
Dec 12, 2011
 |  BioCentury  |  Product Development

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Roche picks PTC's small molecule for spinal muscular atrophy

A second deal between Roche and PTC Therapeutics Inc. demonstrates both the pharma's increasing push into CNS diseases, and its confidence in PTC's small molecule approaches to modifying RNA biology.

Last month, the partners announced Roche licensed exclusive rights to a preclinical program for spinal muscular atrophy (SMA) in exchange for $30 million up front, up to $460 million in milestones and potential double-digit royalties.

Under a 2009 deal, the partners already were using PTC's Gene Expression Modulation by Small-Molecules (GEMS) technology to discover and develop compounds against four undisclosed CNS targets. Those programs are in preclinical development.

GEMS identifies small molecules that modify the composition and activity of the untranslated regions of mRNA to either increase or decrease the level of protein production.

The SMA program was discovered using a different screening technology PTC calls "alternate splicing." It identifies compounds that drive a desired...

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