Pipeline



With science and patients at the center of what we do, Sanofi Genzyme continues to focus on developing scientific approaches and novel therapies for complex and debilitating diseases. Explore our pipeline to see highlights of our research programs in various stages of clinical development.

Last Updated: January 19, 2016

Items marked with a ** are being developed in collaboration with Alnylam® and *** are being developed with Regulus 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
Patisiran**
Familial Amyloid Polyneuropathy
RNAi
Phase III
Revusiran**
Familial Amyloid Cardiomyopathy
RNAi
Phase III
Olipudase (acid sphingomyelinase)
Niemann-Pick disease Type B
protein-based therapy
Phase II
NeoGAA GZ402666
Pompe disease, 2nd generation enzyme
protein-based therapy
Phase I
GZ402671
Fabry disease
small molecule
Phase II
SAR339375 (RG-012)***
Alport Syndrome
oligonucleotide
Phase I
SAR422459
Stargadrt disease

gene therapy

Phase I
Fitisuran**
Hemophilia
RNAi
Phase I
GZ389988
Osteoarthritis

small molecule

Phase I
SAR366234
Elevated intraocular pressure

small molecule

Phase I
SAR421869
Usher syndrome 1B

gene therapy

Phase I
Multiple Sclerosis
GZ402668
Multiple Sclerosis
mAb
Phase I

The agents mentioned here are investigational and have not been approved by the US Food and Drug Administration (FDA) or any other regulatory agency worldwide for the uses under investigation.

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 Lebers congenital amaurosis type 1. We have several programs in multiple sclerosis with molecules addressing areas of unmet need, using approaches such as immunomodulation, neuroprotection, and remylination.

Last Updated: 3/17/2016
GZUS-GZ-16-02-0564
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