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news.PTC Therapeutics and Genzyme have made changes in their existing collaboration agreement, under which PTC has regained worldwide rights to ataluren.
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Discovered by PTC, ataluren is an experimental protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
The tie up also allows Genzyme to retain an option for commercializing ataluren in indications other than nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) outside the US and Canada.
Under the agreement, PTC is expected to conduct worldwide development in all indications with a near-term focus on late-stage programs in nmDBMD and nonsense mutation cystic fibrosis.
Genzyme chief operating officer David Meeker said the decision to restructure this agreement is a result of a recent portfolio assessment, and will enable them to intensify their focus on other areas of development within Genzyme.