Pipeline
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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: March 28, 2013

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

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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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Eliglustat tartrate
Gaucher disease
Small Molecule
Fabrazyme® (agalsidase beta)
Fabry disease, label expansion
Protein-Based Therapy
Acid sphingomyelinase
Niemann-Pick disease type B
Protein-Based Therapy
AAV-hAADC gene therapy
Parkinson's disease
Gene Therapy
AAV-sFLT gene therapy
Age-related macular degeneration
Gene Therapy
Neo-rhGAA
Pompe disease, 2nd generation enzyme
Protein-Based Therapy
Genz-682452
Fabry disease, substrate reduction
Small Molecule

Multiple Sclerosis

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Alemtuzumab*
Multiple sclerosis
Protein-Based Therapy

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