The cooperative research and development agreement with the National Institutes of Health (NIH) gives Amsterdam Molecular Therapeutics (AMT) the option to license the exclusive rights to the recombinant adeno-associated virus baculoviral manufacturing technology developed for treating Duchenne muscular dystrophy (DMD) by Robert Kotin and colleagues at the National Heart, Lung, and Blood Institute (NHLBI) Laboratory of Biochemical Genetics.
While AMT’s current production platform is based on a 50-liter bioreactor set-up, the application of the NIH technology has been scaled-up to 250 liters, and allows for further expansion to 1,000 liters. The scale-up is also important for treatment of Duchenne muscular dystrophy for which AMT has just closed an agreement with La Sapienza University in Rome, Italy, since this indication requires a relatively high dose of gene therapy vector in order to correct the defect in all affected muscles.
Ronald Lorijn, CEO of AMT, said: “This cooperative research and development agreement with the NIH provides AMT with a technology that in combination with our proprietary in-house platform adds a whole new dimension to our manufacturing platform.”