12:00 AM
Jul 21, 2008
 |  BioCentury  |  Strategy

PTC's no nonsense deal

Two decades ago, researchers thought cystic fibrosis would be one of the easier diseases to cure: simply fix the CFTR gene defect and that would be that. Fast forward 20 years, and there are no disease-modifying drugs for CF. Genzyme Corp., which has worked in this area through much of the intervening period, thinks it may have found a disease modifier in PTC124, a small molecule from PTC Therapeutics Inc. that is in Phase II testing for CF and muscular dystrophy.

Thanks to the agent’s disease modifying potential across those previously untreatable disorders, PTC was able to command $100 million up front while retaining U.S. and Canadian commercialization rights. Genzyme clearly didn’t mind the price tag or territories, and expects the compound will fit into its portfolio of orphan drugs for genetic diseases.

PTC124 targets nonsense mutations to induce ribosomal read-through of the single-point alterations in the genetic code and avoid premature protein translation. PTC believes the...

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