Research and Pipeline

Vertex is focused on discovering, developing and commercializing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.

These studies are investigating treatments or outcomes that have not received approval from a health authority. The information presented is not intended to convey conclusions of safety or efficacy. There is no guarantee that the outcome of these studies will result in approval by a health authority.

Cystic Fibrosis (CF)

Ivacaftor
Phase 4
Lumacaftor/Ivacaftor
Phase 4
Tezacaftor/Ivacaftor + Ivacaftor
Phase 4
ELEXACAFTOR/TEZACAFTOR/IVACAFTOR + IVACAFTOR
Phase 4
VX-561
Phase 2
VX-121 + TEZACAFTOR + VX-561 
Phase 2

Pain

VX-150
Phase 2

Hemoglobinopathies

BETA THALASSEMIA – CTX001
Phase 1/2
SICKLE CELL DISEASE – CTX001
Phase 1/2

Alpha-1 Antitrypsin Deficiency

VX-814
Phase 2
VX-864
Phase 1

Duchenne Muscular Dystrophy (DMD)

DUCHENNE MUSCULAR DYSTROPHY

Outlicensed

VX-787 (OUTLICENSED TO JANSSEN PHARMACEUTICALS, INC.)
Phase 3
VX-970 (OUTLICENSED TO MERCK KGAA, DARMSTADT, GERMANY)
Phase 2
VX-803 (OUTLICENSED TO MERCK KGAA, DARMSTADT, GERMANY)
Phase 1
VX-984 (OUTLICENSED TO MERCK KGAA, DARMSTADT, GERMANY)
Phase 1