ARTICLE | Product Development

Checking the boxes in gene therapy

February 8, 2010 8:00 AM UTC

Amsterdam Molecular Therapeutics thinks its Glybera represents the right combination of vector, gene and disease setting to become the first gene therapy to be approved outside of China.

The European Medicines Agency now has accepted an MAA for the product to treat lipoprotein lipase deficiency. The company hopes a positive decision early next year will validate its adeno-associated virus (AAV) vector technology and boost interest from potential partners in the rest of its pipeline...