Genzyme's R&D efforts span a range of clinical needs. Often our research centers on rare, or orphan, diseases that get little attention elsewhere. Other times it's in more widely studied medical areas, but focused on a specific niche or novel treatment approach.
We have particularly strong research programs in the medical areas that correspond with our business units – rare diseases and neuroimmunological disorders such as multiple sclerosis. We are on the forefront of solving medical challenges within these therapeutic areas. For example, most medicines that treat lysosomal storage disorders (LSDs) are infusions that can’t treat the iterations of these diseases that affect the central nervous system (CNS). Our scientists are currently investigating a small molecule for Fabry disease that has the potential to reach the CNS. If successful, this approach could be used with other diseases and could revolutionize the treatment of neuropathic LSDs for a group of patients who had no treatment options.
Within neuroimmunology, we are already starting on next-generation MS treatments. Our goal is to find treatments that not only stop disease progression but also repair the damaged immune system, and we are researching a variety of technology platforms – including small molecules and antibodies – to accomplish this goal.
We also pursue promising research outside our defined areas of focus; these product candidates may eventually be commercialized by another part of Sanofi.