The brief

Analyst view

• Vertex's CF franchise (Kalydeco, Trikafta) is the market leader; Casgevy is the first approved CRISPR therapy.
• Revenue in 2025 reached $12 billion; net income $3.95 billion; CEO Reshma Kewalramani.
• Zimislecel for type 1 diabetes showed 83% insulin independence in early trials; Phase 3 enrollment complete, BLA expected 2026.

Technical approach

As reported

Rational design

Structure-based drug discovery

Vertex pioneered computational design: using protein structures to engineer compounds that inhibit disease targets, not random library screening.

Gene editing

CRISPR/Cas9 cell therapy

Casgevy edits blood stem cells to protect red cells from sickling, then returns them to cure sickle cell disease without ongoing treatment.

Cell therapy

Stem-cell-derived islets

Zimislecel grows insulin-producing islets from stem cells, eliminating donor organs and enabling an off-the-shelf cure for type 1 diabetes.