Gene therapy firm Amsterdam Molecular Therapeutics (AMT) negotiated an option to license a gene therapy candidate for the lysosomal storage disease Sanfilippo B, as part of a deal through which it will also carry out the cGMP manufacture of the product for a Phase I/II study. The treatment is in development by an Institut Pasteur-led French Consortium, with funding up to the end of a Phase I/II study pledged by the French muscular dystrophy association (AFM). The aim is to develop a gene therapy to restore the alpha-N-acetylglucosaminidase enzyme lacking in the brain cells of Sanfilippo B patients.
AFM is also funding the manufacturing costs, and will pay AMT Euro 1.8 million for its contractual work to produce and supply the adeno-associated viral 5 (AAV5) gene therapy product to the consortium. Institut Pasteur will lead the development program and carry out the initial Phase I/II clinical trial, which is expected to start during 2013. At the end of this study AMT will have an option to acquire full commercial rights to the therapy.
“AMT is one of the only companies in the world that has a proven ability in manufacturing cGMP quality gene therapy products, not only in batches sufficient for clinical development, but also in support of a potential regulatory approval,” remarks Muriel Eliaszewicz, M.D., Institut Pasteur’s medical director.
In December 2010 the consortium published preclinical data demonstrating the safety of the Sanfilippo B gene therapy product and its ability to spread throughout the brain in models of the disease. The studies, published in Molecular Therapy, also showed that the administered gene vectors exhibited long-term viability within cells and led to an improvement in histological and biochemical markers.