Our lab is focused on developing therapies for some of the world’s most devastating muscular dystrophies and neuromuscular diseases including DM1, FSHD and ALS. We employ state of the art technologies in order to dissect the pathways and underlying causes of disease pathology as a method of gaining insight into ways of treating disorders and then develop the medicines to help patients. Modeling of these diseases is critical to our success and through patient derived cellular systems and in vivo models, we craft molecular and pathological readouts that enable the drug discovery efforts. Novartis is able to harness a vast array of therapeutic options including small molecules, biologics as well as gene therapies. With over 20 years of experience in both biotechnology and pharmaceutical drug development I have learned to generate creative solutions to answer fundamental questions of biology and our lab is supported by a vast infrastructure of groups dedicated to next generation sequencing, genetic and small molecule screening, protein analytics and transgenic animal development.
For many of the genetically caused disorders within our focus, gene therapy may provide an effective treatment and even a cure. We are exploring such therapies using AAV mediated delivery. In addition to identifying and developing therapeutic molecules suitable for delivery to patients, we are engaged in the study of payload packaging and capsid engineering aimed at developing more effective ways of delivering these life-saving medicines.