What is Vertex Pharmaceuticals?
Transformative medicines for cystic fibrosis, sickle cell disease and beta thalassemia
HQ Full Address
Boston, Massachusetts, United States
Revenue Growth Rate
10.51%
Product Features & Capabilities
- Investigational therapies for cystic fibrosis
- therapies for sickle cell disease
- treatments for transfusion-dependent beta thalassemia
- investigational cell therapy for type 1 diabetes
- investigational therapies for APOL1-mediated kidney disease
- investigational pain management solutions
Investment Focus
Vertex Pharmaceuticals primarily focuses on therapeutic areas including cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia. They are also involved in research and development for other serious diseases, such as type 1 diabetes and various kidney diseases.
Other Considerations
Recognized as a top workplace by Science Magazine, The Boston Globe, and Boston Business Journal; Named on TIME Most Influential Companies and Fast Company Most Innovative Companies lists; Awarded 2025 Lasker-DeBakey Clinical Medical Research Award; Earned Breakthrough Prize and Prix Galien recognitions; Has clinical trial data for investigational therapies in type 1 diabetes and kidney diseases
Latest Funding Round
Vertex Pharmaceuticals completed a Post-IPO Secondary funding round on July 10, 2024, raising approximately $1,111,135.
Awards Recognition
Vertex Pharmaceuticals has received several notable awards and recognitions in the biotech field, including:
1. The 2024 Breakthrough Prize in Life Sciences awarded to three of its researchers for developing life-transforming drug combinations.
2. Recognition from MassBio, where Vertex Pharmaceuticals was honored alongside other companies for its contributions to the biotech industry.
3. The 2023 Distinguished Alumni Award received by CEO Reshma Kewalramani from Boston University.
Key Innovations
- KAFTRIO® (elexacaftor/tezacaftor/ivacaftor) - A combination therapy for cystic fibrosis that targets the underlying cause of the disease.
- KALYDECO® (ivacaftor) - The first drug approved to treat the underlying cause of cystic fibrosis in patients with specific mutations.
- CASGEVY® (exagamglogene autotemcel) - A gene therapy for sickle cell disease that utilizes CRISPR technology to edit patients' blood-forming stem cells.
- JOURNAVX™ (suzetrigine) - A non-opioid pain reliever aimed at providing alternatives to traditional pain management.
- VX-522 - An investigational therapy for cystic fibrosis currently in development.
Vertex is also investing in cell and genetic therapies, expanding their portfolio to include treatments for other conditions such as type 1 diabetes and pain management.
Regulatory Approvals
- CASGEVY™ (exagamglogene autotemcel) - Approved by the U.S. FDA for the treatment of sickle cell disease in December 2023.
- TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor) - The FDA approved the expanded use of TRIKAFTA to include children with cystic fibrosis aged 6 to 11 years in April 2023.
- Journavx (suzetrigine) - A non-opioid pain medication that received FDA approval as a safer alternative to addictive opioids in early 2024.
- Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) - Recently approved for cystic fibrosis treatment, providing another option for patients.
Partnerships
- Collaboration with Orna Therapeutics - Vertex partnered with Orna Therapeutics in a $4 billion collaboration to develop next-generation gene therapies for sickle cell disease and beta-thalassemia.
- Collaboration with CRISPR Therapeutics - In March 2023, Vertex announced a licensing agreement with CRISPR Therapeutics, paying $100 million for non-exclusive rights to their technology for developing hypoimmune gene-edited therapies.
- Partnership with Lonza - Vertex is collaborating with Lonza to develop a new state-of-the-art manufacturing facility in Portsmouth, NH, which will enhance their production capabilities.
- Collaboration with Stablix - Vertex and Stablix established a strategic collaboration in April 2023 to discover and develop targeted protein stabilization therapeutics.
- Expansion with Genomics - Vertex expanded its collaboration with Genomics to accelerate the discovery and development of precision medicines.