Amsterdam Molecular Therapeutics (AMT) amended its license agreement with Amgen for gene therapy applications incorporating the GDNF (glial cell-derived neurotrophic factor) gene, to which Amgen holds rights. AMT will now be able to leverage GDNF in gene therapies for a range of CNS applications as well as to look for development partners.
The original deal, inked in September 2008, allowed AMT to use the GDNF gene to treat Parkinson disease. The gene contains information to produce a protein necessary for the development and survival of nerve cells. Studies with a GDNF gene therapy, AMT-090, in a Parkinson disease model are being conducted by AMT in collaboration with the University of Lund, Sweden.
AMT will now look toward combining the GDNF gene with its adeno-associated virus (AAV) technology to develop gene therapies for a other CNS diseases like Huntington disease and amyotrophic lateral sclerosis (ALS). The aim is to protect and enhance the function of the affected nerve cells.
“Based on the promising early results of our GDNF gene therapy product in Parkinson disease models, we believe there is an opportunity for a similar approach in other debilitating CNS disorders,” states Jorn Aldag, CEO of AMT.
“This agreement will allow us to progress the program for Parkinson disease forward and at the same time find a partner who will support the funding of our GDNF programs in alternative indications. Expanding the partnering opportunities could mean even greater interest as the widened therapeutic applications offer more chances of success, potentially less complex product development paths, and in many cases fewer patients to enroll in clinical trials.”
AMT concentrates on developing human gene-based therapies. The company says that it has designed and validated a stable and scalable AAV production platform.
AMT’s lead product, Glybera, is for the treatment of lipoprotein lipase deficiency. The company has already filed for approval in the EU. In the U.S. the drug is in Phase II/III trials. Additionally, AMT has a Phase I candidate for hemophilia B and preclinical products for Duchenne muscular dystrophy, acute intermittent porphyria, and Parkinson disease.