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Orphan Drug Company Sarepta Therapeutics’ Competitor Has New Stategic Advisor

December 17, 2012

Henri Termeer, former Genzyme CEO and Chairman who retired in 2011 after almost 30 years, joins Prosensa as a strategic adviser “providing advice for the biotech’s corporate strategy and growth plans’’. Mr. Termeer built Genzyme into a leader in rare diseases and orphan drugs. His joining Prosensa comes as Prosensa’s drug that treats Duchenne Muscular Dystrophy (DMD) is in late-stage development with partner GlaxoSmithKline.

Could this be a threat to Sarepta Therapeutics ?  Prosensa’s drug is a competitor of Sarepta Therapeutics’ orphan drug Eteplirsen for DMD. Prosensa has RNA therapies for different variants of DMD.

Prosensa’s orphan drug candidate PRO051 specifically “induces exon 51 skipping in the DMD gene, which, given the frequencies in various international DMD mutation databases, could in principle correct the reading frame in approximately 13% of all DMD patients …’’.  PRO051 has orphan drug designation in both the EU and the US.

References December 13, 2012 article titled, Ëx-Genzyme CEO Termeer returns to his roots at biotech Prosensa”

Prosensa December 13, 2012 Press Release

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