Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.
Genzyme is in the early stages of investigating a next generation enzyme replacement therapy (neoGAA) for Pompe disease. It differs from the original ERT, whereby carbohydrates are bonded onto the enzyme during the complex manufacturing process of neoGAA. These carbohydrates are thought to help the enzyme enter the muscles more efficiently. Though it’s still very early in the development process and there are many unknowns, the hope is that through this more efficient uptake into the muscle cell, neoGAA might improve on the effectiveness of the existing therapy.
An article published on PubMed.gov, Dec. 2011, describes a 40-year old woman with Pompe Disease who successfully carried a healthy baby to term.