Vertex Pharmaceuticals Incorporated

Introduction
Vertex Pharmaceuticals is a global biotechnology company focused on discovering and developing transformative medicines for serious diseases. Founded in 1989, Vertex is best known for pioneering treatments for cystic fibrosis (CF), becoming the first company to develop medicines that target the underlying cause of the disease rather than just its symptoms. Beyond CF, Vertex is expanding into gene editing, rare diseases, pain, and other therapeutic areas.

Mission and Vision

Mission: To discover, develop, and deliver transformative medicines for people with serious diseases.
Vision: To be a global leader in scientific innovation, advancing cures and life-changing therapies that address unmet medical needs.

Products and Services

Approved Therapies:
- Trikafta/Kaftrio (triple combination therapy for CF)
- Symdeko, Orkambi, Kalydeco (earlier CF treatments)
Research & Development:
- Gene editing therapies in collaboration with CRISPR Therapeutics (e.g., exa-cel for sickle cell disease & beta thalassemia).
- Pipeline in kidney disease, type 1 diabetes, pain, and other rare genetic disorders.
Scientific Focus: Precision medicine, genetic therapies, and small molecule innovation.

Growth and Innovation
Vertex revolutionized cystic fibrosis care, turning a fatal childhood disease into a manageable condition for many patients. The company continues to grow by expanding into gene-editing therapies (CRISPR-based), regenerative medicine, and novel small molecules. Strategic collaborations with companies like CRISPR Therapeutics, Moderna, and Arbor Biotechnologies highlight its role as a pioneer in next-generation biotechnology.

Global Impact
Vertex medicines are available in more than 50 countries, improving the lives of tens of thousands of CF patients. Its gene-editing programs have the potential to provide functional cures for genetic blood disorders. Vertex also supports patient communities and invests in STEM education to inspire future generations of scientists.