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A collaboration worth up to $2.65B that produced Casgevy—the first CRISPR-based gene therapy approved by the FDA. Vertex's development machine plus CRISPR Therapeutics' gene-editing platform delivered a functional cure for sickle cell disease, marking when gene editing became clinical reality.
Vertex and CRISPR Therapeutics entered a strategic collaboration to use CRISPR/Cas9 gene editing for hemoglobinopathies (sickle cell disease and beta-thalassemia). This led to the development and 2023 FDA approval of Casgevy (exagamglogene autotemcel), the first CRISPR-based gene therapy approved in the US.
2K US cases/yr · $1.5B Projected SCD Gene Therapy Market (2025)
Casgevy uses CRISPR/Cas9 to edit patients' stem cells, reactivating fetal hemoglobin to prevent sickling. It was the first CRISPR-based gene therapy approved by the FDA (Dec 2023).
Casgevy (exa-cel, Vertex/CRISPR) competes directly with Lyfgenia (lovo-cel, bluebird bio), a lentiviral gene therapy approved same-day (Dec 2023). Non-gene-therapy SCD options include hydroxyurea, Oxbryta (voxelotor, Pfizer; withdrawn 2024), Adakveo (crizanlizumab, Novartis), and Endari (L-glutamine, Emmaus Medical). Stem cell transplant remains the only other curative option.
300 US cases/yr · $600M Projected TDT Gene Therapy Market (2025) · 15-20 Annual Transfusions for TDT Patients
Transfusion-dependent beta-thalassemia requires lifelong blood transfusions and iron chelation. Casgevy offers a potential one-time functional cure through CRISPR editing of the BCL11A erythroid enhancer.
Casgevy competes with Bluebird Bio's Zynteglo (lentiviral gene therapy for TDT, approved 2022). Both require myeloablative conditioning.
Vertex and CRISPR Therapeutics partnered on CRISPR/Cas9 gene editing for hemoglobinopathies (SCD, beta-thal).
FDA approved Casgevy for SCD and TDT. First CRISPR-based gene-editing therapy ever approved. $200M milestone paid.
First CRISPR therapy approved. Vertex leads commercialization (60/40 split). Early commercial ramp ($10M 2024 sales).
| Deal | Year | Value | Outcome |
|---|---|---|---|
| Vertex Pharmaceuticals Incorporated / CRISPR Therapeutics AG (this deal) | 2015 | $2.6B | 85 |
| Vertex Pharmaceuticals Incorporated / Aurora Biosciences Corporation | 2001 | $592M | 95 |
| Vertex Pharmaceuticals Incorporated / Semma Therapeutics Inc. | 2019 | $950M | 86 |
| Vertex Pharmaceuticals Incorporated / Concert Pharmaceuticals, Inc. | 2017 | $250M | 85 |
| Vertex Pharmaceuticals Incorporated / Alpine Immune Sciences | 2024 | $4.9B | 78 |
| Vertex Pharmaceuticals Incorporated / Crinetics Pharmaceuticals, Inc. | 2026 | $10.0B | 68 |
| Vertex Pharmaceuticals Incorporated / ViaCyte Inc. | 2022 | $320M | 60 |
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More: 2015 deals · Vertex Pharmaceuticals Incorporated deals · Gene Therapy deals