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: March 5, 2015

Items marked with a ** are being developed in collaboration with Alnylam and * are being developed in collaboration with Voyager Therapeutics.

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.
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

Expand
Patisiran**
Familial amyloid polyneuropathy
RNAi
Phase III
Revusiran**
Familial amyloid cardiomyopathy
RNAi
Phase III
GZ402671
Fabry disease, substrate reduction
small molecule
Phase II
GZ402671
Gaucher disease Type 3, substrate reduction
small molecule
Phase I
Olipudase (acid sphingomyelinase)
Niemann-Pick disease type B
protein-based therapy
Phase I
NeoGAA GZ402666
Pompe disease, 2nd generation enzyme
protein-based therapy
Phase I
AAV2-hAADC*
Parkinson's disease
gene therapy
Phase I

Multiple Sclerosis

Expand
Vatelizumab
Multiple Sclerosis
mAb
Phase II
GZ402668
Multiple Sclerosis
mAb
Phase I

In addition to the candidates in clinical development, we are conducting earlier stage research in a range of therapeutic areas including genetic diseases such as cystic fibrosis, spinal muscular atrophy, and Leber’s congenital amaurosis type 1. We have several promising programs in multiple sclerosis with molecules targeting aspects of the disease that current therapies do not adequately address, using approaches such as immunomodulation, neuroprotection, and remyelination.

Last Updated: 4/1/2015
infoBox