FDA Approves First Landmark CRISPR Gene-Editing Therapy for Sickle Cell Disease
A historic milestone in medicine as the FDA clears the first-ever CRISPR-based gene-editing treatment, marking a new era for genomic medicine.
Vertex Pharmaceuticals
Vertex Pharmaceuticals is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. Vertex is the leading developer of cystic fibrosis therapies (Trikafta/Kaftrio franchise), brought the first FDA-approved CRISPR-based therapy to market (Casgevy, with CRISPR Therapeutics), and has a late-stage pipeline in acute pain, sickle cell disease, beta thalassemia, and Duchenne muscular dystrophy.
Company Info
Sector: Biotechnology / Therapeutics
Headquarters: Boston, Massachusetts, USA
Founded: 1989
Website: www.vrtx.com/
Upcoming Milestones & Key Dates
Q2 2026
Earnings
Q1 2026 Earnings — Trikafta franchise and Casgevy launch update
Mid-2026
Launch
Commercial rollout and formulary coverage updates for Journavx (suzetrigine) in acute pain
November 18, 2026
Conference
ASH Annual Meeting — Casgevy real-world and pipeline data
Clinical Development Pipeline
Casgevy (exagamglogene autotemcel)
Target: BCL11A / CRISPR-Cas9 gene editing (with CRISPR Therapeutics)Trikafta / Kaftrio (elexacaftor-tezacaftor-ivacaftor)
Target: CFTR corrector + potentiatorJournavx (suzetrigine)
Target: NaV1.8 sodium channel inhibitorRecent News
Leadership Team
Careers & Job Openings