In July, Amsterdam Molecular Therapeutics (AMT), a Netherlands-based company specializing in human gene therapy, announced its collaboration with St. Jude Children’s Research Hospital in Memphis, TN, to develop a gene therapy for hemophilia B patients. The treatment will use AMT’s proprietary adeno-associated virus (AAV) vectors, which are employed as vehicles for delivering the targeted therapeutic genes.
The drug development research will be conducted at St. Jude’s, while AMT will hold exclusive commercial rights to the final product.
AMT’s unique gene therapy platform appears to circumvent some of the obstacles that have prevented gene therapy success in the past. The goal is to cure patients of hemophilia B with a single administration of the product. Many patients currently self-infuse the clotting factor up to three times weekly.
“The importance of this collaboration with renowned St. Jude Children’s Research Hospital stretches beyond its scientific and business aspects. This collaboration will bring to these patients the hope that a real cure is on the horizon,” said Ronald Lorijn, CEO of AMT.
Source: AMT news release dated July 15, 2008