Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, has successfully treated Duchenne muscular dystrophy (DMD) in an animal model with its proprietary gene therapy. The proof of concept studies were performed in collaboration with the group of Professor Irene Bozzoni (University of Rome, La Sapienza, Italy) and demonstrated effectiveness in the heart as well as in skeletal muscles. In a previous study, AMT's gene therapy approach was shown to be successful in the treatment of diseased human muscle cells obtained from biopsies of DMD patients. These data establish a robust basis for AMT's therapeutic approach to DMD.
"We are proud to establish proof of concept with our gene therapy for Duchenne muscular dystrophy, as it is an important new step in developing a treatment for this progressive and devastating disease," said Jörn Aldag, chief executive officer of AMT. "In particular, our positive results in the heart and respiratory muscles bring hope that we might be able to prevent the fatal outcome of this disease in the future, potentially through a long-term remedy with a single treatment."
AMT has a unique gene therapy platform that appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine.