Vertex Pharmaceuticals Incorporated
Global biotechnology company focused on discovering, developing and producing medicines for serious diseases. Core activities include small-molecule drug discovery, biologics and cell & gene therapy research, clinical development, manufacturing scale‑up for advanced therapeutics, and patient support and access programs. The company operates multiple research, laboratory and manufacturing sites worldwide and reports active corporate responsibility and sustainability programs.
Industries
Nr. of Employees
Very Large (1000+)
Vertex Pharmaceuticals Incorporated
Patents
7- or 8-hydroxy-isoquinoline and 7- or 8-hydroxy-quinoline derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD)
US-12662471-B2
View DetailsHydroxy and (halo)alkoxy substituted tetrahydrofurans as modulators of sodium channels
US-12662470-B2
View DetailsCompositions and methods for editing beta-globin for treatment of hemaglobinopathies
US-12653860-B2
View Details
7- or 8-hydroxy-isoquinoline and 7- or 8-hydroxy-quinoline derivatives as alpha-1-antitrypsin modulators for treating alpha-1-antitrypsin deficiency (AATD)
US-12662471-B2
View DetailsHydroxy and (halo)alkoxy substituted tetrahydrofurans as modulators of sodium channels
US-12662470-B2
View DetailsCompositions and methods for editing beta-globin for treatment of hemaglobinopathies
US-12653860-B2
View DetailsProducts
CFTR modulator therapies for cystic fibrosis
Orally administered small-molecule modulators designed to improve CFTR protein function to address the underlying cause of cystic fibrosis.
Autologous ex vivo gene-edited therapy for hemoglobinopathies
An autologous ex vivo CRISPR/Cas9 gene-editing therapy developed and advanced into clinical use for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia.
Stem cell–derived islet cell therapy candidates for type 1 diabetes
Allogeneic stem cell–derived, fully differentiated insulin-producing cell therapies under clinical investigation, delivered by infusion or via implanted encapsulation devices.
Small-molecule inhibitors for APOL1-mediated kidney disease
Small-molecule programs aimed at inhibiting APOL1 protein function to address underlying causes of APOL1-mediated kidney diseases.
Non-opioid acute pain therapies and support programs
Development and commercialization of non-opioid acute pain medicines accompanied by patient support services to facilitate access and appropriate use in acute care settings.
ALYFTREK
ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) is a fixed-dose combination film-coated tablet containing a CFTR corrector and potentiator indicated for the treatment of cystic fibrosis in patients aged 6 years and older with at least one F508del mutation or another responsive mutation.
CFTR modulator therapies for cystic fibrosis
Orally administered small-molecule modulators designed to improve CFTR protein function to address the underlying cause of cystic fibrosis.
Autologous ex vivo gene-edited therapy for hemoglobinopathies
An autologous ex vivo CRISPR/Cas9 gene-editing therapy developed and advanced into clinical use for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia.
Stem cell–derived islet cell therapy candidates for type 1 diabetes
Allogeneic stem cell–derived, fully differentiated insulin-producing cell therapies under clinical investigation, delivered by infusion or via implanted encapsulation devices.
Small-molecule inhibitors for APOL1-mediated kidney disease
Small-molecule programs aimed at inhibiting APOL1 protein function to address underlying causes of APOL1-mediated kidney diseases.
Non-opioid acute pain therapies and support programs
Development and commercialization of non-opioid acute pain medicines accompanied by patient support services to facilitate access and appropriate use in acute care settings.
ALYFTREK
ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) is a fixed-dose combination film-coated tablet containing a CFTR corrector and potentiator indicated for the treatment of cystic fibrosis in patients aged 6 years and older with at least one F508del mutation or another responsive mutation.
Services
Funding and drug support for independent clinical, observational and nonclinical research aligned to company disease areas of interest.
Personalized support services that provide education, care coordination and insurance navigation for patients prescribed advanced therapies.
Funding and drug support for independent clinical, observational and nonclinical research aligned to company disease areas of interest.
Personalized support services that provide education, care coordination and insurance navigation for patients prescribed advanced therapies.
Expertise Areas
- Small-molecule drug discovery
- Cell and gene therapy development
- Gene editing (ex vivo and in vivo)
- Clinical trial design and global execution
Key Technologies
- CRISPR/Cas9 gene editing
- Ex vivo gene-editing workflows
- In vivo gene editing approaches
- Stem cell–derived cell therapy
News & Updates
Presentation of Phase 3 RAINIER data supporting potential best-in-class profile for an investigational program in IgA nephropathy.
Announcement of positive Phase 2b results and completion of enrollment in the Phase 2/3 AMPLITUDE trial for an investigational program in APOL1-mediated kidney disease.
Announcement that an external clinical-stage company has been acquired and integrated into the company’s portfolio.
Named by Fast Company as one of the Most Innovative Companies for 2026.
Included on Fortune’s 100 Best Companies to Work For list in 2026.
Recognized by Forbes as one of the World's Best Employers.
Presentation of Phase 3 RAINIER data supporting potential best-in-class profile for an investigational program in IgA nephropathy.
Announcement of positive Phase 2b results and completion of enrollment in the Phase 2/3 AMPLITUDE trial for an investigational program in APOL1-mediated kidney disease.
Announcement that an external clinical-stage company has been acquired and integrated into the company’s portfolio.
Named by Fast Company as one of the Most Innovative Companies for 2026.
Included on Fortune’s 100 Best Companies to Work For list in 2026.
Recognized by Forbes as one of the World's Best Employers.