NASDAQ: VRTX

The Science of Possibility

Investing in scientific innovation to create transformative medicines for people with serious diseases. From cystic fibrosis to sickle cell disease, we're redefining what's possible in medicine.

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Scientist in laboratory

Transforming Lives Since 1989

Founded in Cambridge, Massachusetts, Vertex Pharmaceuticals is one of the first biotech firms to use rational drug design. Today, headquartered in Boston's Seaport District, we continue to pioneer therapies for serious diseases.

With over 6,400 employees worldwide and research facilities in Boston, San Diego, and Oxford, we are committed to discovering, developing, and producing innovative medicines that help people lead better lives.

35+ Years of Innovation
6,400+ Employees Worldwide
60+ Countries Reached

Our Science

Focused on discovering and developing innovative medicines across multiple therapeutic areas

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Cystic Fibrosis

Pioneering CFTR modulators including Trikafta, the first triple-combination therapy treating up to 90% of CF patients with at least one F508del mutation.

Market Leader
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Sickle Cell & Beta Thalassemia

CASGEVY, our CRISPR/Cas9 gene-edited therapy, represents a potential functional cure for patients with these serious blood disorders.

Gene Editing
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Acute Pain

Journavx (suzetrigine) is a novel oral non-opioid treatment for moderate to severe acute pain—the first new pain medicine type in 25 years.

FDA Approved
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APOL1-Mediated Kidney Disease

Developing potential therapies for IgA nephropathy and other serious kidney diseases with high unmet medical need.

Clinical Stage
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Type 1 Diabetes

Through our acquisition of ViaCyte, we're advancing VX-880 and stem cell-derived islet cell therapies as potential curative approaches.

Cell Therapy
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Alpha-1 Antitrypsin Deficiency

Investigating small molecule correctors to address the underlying genetic cause of this rare liver and lung disease.

Research

Our Medicines

Transformative therapies that have changed the standard of care for serious diseases

Trikafta

Cystic Fibrosis

Triple-combination CFTR modulator (elexacaftor/tezacaftor/ivacaftor) approved for patients 12+ with at least one F508del mutation, covering approximately 90% of the CF population.

CASGEVY

Sickle Cell & Beta Thalassemia

Exagamglogene autotemcel—the first CRISPR/Cas9 gene-edited therapy approved for transfusion-dependent beta thalassemia and severe sickle cell disease.

Journavx

Acute Pain

Suzetrigine, a novel oral non-opioid NaV1.8 inhibitor for moderate to severe acute pain in adults, offering an alternative to opioid-based management.

Kalydeco

Cystic Fibrosis

Ivacaftor, the first medicine to treat the underlying cause of CF rather than just symptoms, for patients with specific CFTR gene mutations.

Orkambi

Cystic Fibrosis

Lumacaftor/ivacaftor combination for CF patients ages 1+ with two copies of the F508del mutation in the CFTR gene.

Alyftrek

Cystic Fibrosis

Vanza triple-combination therapy expanding treatment options for CF patients with rare mutations, approved with label extensions in 2026.

Global Impact

Working to ensure patients worldwide have access to transformative medicines

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60+ Countries

Our CF medicines are broadly available in over 60 countries worldwide, with reimbursement agreements in 36+ countries.

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340+ Clinical Trials

Robust clinical development program advancing our pipeline across multiple therapeutic areas and patient populations.

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Patient Support

Comprehensive access programs and patient support services to help eligible patients start and stay on treatment.

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Vertex Foundation

Supporting underserved communities, STEM education, and the sickle cell disease community through strategic grants and partnerships.

Join Us in Transforming Lives

Whether you're a patient, healthcare provider, researcher, or potential team member, we invite you to be part of our mission.

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