Research Areas

Many of us here at Genzyme can put faces to the diseases we are working on. For most of us, research is more than just an interesting job—it's a personal quest.
— Johanne Kaplan
V.P. of Neuroimmunology Research

For every product Genzyme launches, every treatment we pursue, the starting point is a significant medical need. We orient our research around therapeutic areas where breakthrough therapies can significantly improve patients' lives.
A Focus on Clinical Needs

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.

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Genetic Disease

Neurologic Disease


Immune-Mediated Disease

Cardiovascular Disease

Renal Disease


A Robust Pipeline of Therapeutic Possibilities

From discovery projects in cystic fibrosis and lupus, to a clinical program for a potential enzyme replacement therapy for Niemann-Pick, to promising phase 3 results for a novel oral therapeutic candidate for Gaucher disease plus much more – our research pipeline demonstrates our abilities to address a range of medical problems.

Many Sources of Inspiration

Our research programs originate from many sources. We continue to build on work in our established therapeutic areas. For example, we pioneered enzyme replacement therapies for lysosomal storage disorders, and today we're investigating small molecule drugs and even gene therapy for those rare genetic disorders. Wherever feasible, we also explore expanding the indications of our existing products, which can lead to potential therapies in an entirely different disease area. For example, our former leukemia antibody product which is now in the Sanofi portfolio, has shown strong clinical study data as a treatment for multiple sclerosis.

Research inspiration can also be as personal as a single scientist's firsthand experience with the effects of a particular disease. We don't limit ourselves to a set list of medical areas, and we're always willing to venture in new directions. At Genzyme, an innovative spirit encourages our researchers to pursue many avenues – while an integrative approach that brings together medical, clinical, regulatory, and commercial experts from throughout the company helps focus our efforts on a realistic end goal.

Applying Innovative Technologies

Our expertise in a variety of medical technology platforms gives us flexibility to find the best possible therapeutic format for the medical challenges we tackle within our research areas.

Last Updated: 5/13/2015
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Genzyme's R&D Brochure

Learn more about the diseases Genzyme is researching, our recent breakthroughs and promising programs, and our long history of successful innovation on behalf of patients.

Newborn Screening for Rare Diseases

Early diagnosis saves and improves lives of individuals with progressive diseases. For years, Genzyme has sponsored research into newborn screening for rare diseases.