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Pfizer Bets On Gene Therapy For Rare Diseases In China

 
• By Brian Yang

After excluding China from its early development plans for domagrozumab, Pfizer’s rare disease R&D team is now gearing to commence a clinical study of the investigational compound on Duchenne muscular dystrophy patients soon.

MUSCULAR DYSTROPHY words written on label tag with medicine,syringe,keyboard and stethoscope
DMD

Despite its potentially large patient pool, China was not initially included in the development plan for PF-06252616 (domagrozumab), a monoclonal antibody that blocks muscular growth inhibiting protein myostatin, when the rare disease team of Pfizer Inc. sat down to plan the study for the investigational compound.

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