Genzyme's R&D efforts span a range of clinical needs. Often our research focuses 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 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. Our scientists are currently investigating a small molecule for Fabry disease that has the potential to reach the central nervous system. 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 working 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 technologies – 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.
Our primary research areas include:
- Genetic Diseases – such as lysosomal storage disorders, cystic fibrosis, myotonic dystrophy, hemophilia, spinal muscular atrophy, Huntington’s disease
- Neurologic Diseases – such as neuropathic LSDs, Parkinson's disease, Huntington's disease, age-related macular degeneration, amyotrophic lateral sclerosis, spinal muscular atrophy
- Neuroimmunology – such as multiple sclerosis, autoimmune neurological disorders
- Immune-Mediated Diseases – such as lupus, giant cell arteritis, type I diabetes, inflammatory bowel disease
- Cardiovascular Diseases – such as severe dyslipidemia, familial hypercholesterolemia, acute myocardial infarction
- Renal Diseases – such as chronic kidney disease, polycystic kidney disease, acute kidney injury
- Orthopedics – such as osteoarthritis, cartilage injury/defects