Pipeline


More on Our Research

Genzyme's full research efforts include far more than what can be featured on this page, from early-stage discoveries to a broad range of clinical trials.

Creating a solid foundation for Genzyme's future growth, our R&D efforts include novel therapeutic candidates as well as new indications for our existing products. Explore our pipeline to see highlights of our most promising research.

Last Updated: January 20, 2015

Items marked with a * have been filed for regulatory review.

Technology The platform or technologic approach upon which a treatment is based; either chemically synthesized like a small molecule or polymer drug, or biologic like a protein, antibody, or cell therapy.
Pre-Clinical

Pre-Clinical testing conducted in the lab and on animals to investigate a treatment’s ability to fight against a targeted disease and to demonstrate safety.

Phase I Clinical trials on small number of human subjects, usually healthy, primarily to assess safety and potential side effects at different dosages.
Phase II Clinical trials on affected patients, primarily to test efficacy; usually randomized and placebo-controlled.
Phase III Clinical trials on affected patients to further test efficacy, benefits, and potential side effects; usually randomized, placebo-controlled, and blinded.

Rare Diseases

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Patisiran
Familial amyloid polyneuropathy
RNAi
Phase III
Revusiran*
Familial amyloid cardiomyopathy
RNAi
Phase III
Olipodase (acid sphingomyelinase)
Niemann-Pick disease type B
protein-based therapy
Phase I
Genz-682452
Fabry disease, substrate reduction
small molecule
Phase II
AAV-sFLT gene therapy
Age-related macular degeneration
gene therapy
Phase I
Neo-GAA
Pompe disease, 2nd generation enzyme
protein-based therapy
Phase I

Multiple Sclerosis

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Vatelizumab
Multiple Sclerosis
protein-based therapy
Phase II
GZ402668
Multiple Sclerosis
protein-based therapy
Phase I

Last Updated: 1/20/2015
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