Explore our pipeline to see highlights of our research programs in various stages of clinical development.
Items marked with ^ are being developed in collaboration with Regeneron and a * are being developed in collaboration with Alnylam®. Items shown with a † denote a Phase 2/3 trial.
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.
Dupilumab^
Eosinophilic esophagitis
mAb
Phase II
Dupilumab^
Nasal Polyposis
mAb
Phase II
Isatuximab
Multiple Myeloma
Phase III
PD-1 Inhibitor^
Cutaneous Squamous Cell Carcinoma
Phase II
Patisiran*
hATTR Amyloidosis
RNAi
Phase III
NeoGAA
Pompe disease, 2nd generation enzyme
protein-based therapy
Phase III
Olipudase Alfa†
Acid Sphingomyelinase Deficiency (ASMD)
protein-based therapy
Phase II
Oral CGS Inhibitor
Fabry disease
small molecule
Phase II
Oral CGS Inhibitor
Parkinson's disease with a
GBA mutation
small molecule
Phase II
Oral CGS Inhibitor
Gaucher Type 3
small molecule
Phase II
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PHASES
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Phase I
Phase II
Phase III
TECHNOLOGIES
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mAb
RNAi
protein-based therapy
small molecule
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: 7/12/2017
GZUS.GZ.16.10.2192(5)