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A $900M bet that paid off in full: Vertex bought an extra 10 points of CTX001 economics from CRISPR Therapeutics, and the asset became Casgevy, the first FDA-approved CRISPR/Cas9 therapy—though CRISPR still gave up 10% of a medicine now approved for both sickle cell disease and beta-thalassemia.
Outcome grade pending — assessed 5 years post-close.
Full analysis, sources & comparables →In two patients (one with transfusion-dependent beta-thalassemia and one with sickle cell disease) treated with autologous CRISPR-Cas9-edited…
Vertex agreed to pay CRISPR Therapeutics $900 million upfront and an additional $200 million upon first regulatory approval to take a larger share of…
The U.S. Food and Drug Administration today approved Casgevy (exagamglogene autotemcel) for the treatment of transfusion-dependent beta-thalassemia in patients…
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Vertex and CRISPR amended their CTX001 collaboration so Vertex leads global development, manufacturing and commercialization with a 60/40 profit/cost split (Vertex 60%). CRISPR received $900M upfront and is eligible for $200M upon first regulatory approval, increasing Vertex's program economics by 10 percentage points.
100K US cases/yr · $3.0B US sickle cell disease + beta-thalassemia drug market 2021 (USD MM, GlobalData) · 2200000 Casgevy US list price per patient (USD)
Sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) are inherited monogenic hemoglobinopathies caused by mutations in the beta-globin gene. SCD affects approximately 100,000 Americans (predominantly Black/African American) and 20 million people globally; TDT affects roughly 1,000-1,500 US patients but >300,000 globally with concentration in Mediterranean, Middle Eastern, and South Asian populations. SCD patients suffer chronic hemolytic anemia, vaso-occlusive crises, end-organ damage (kidney, lung, brain, retina) and reduced life expectancy of 40-50 years. TDT patients require lifelong red blood cell transfusions every 2-5 weeks, leading to iron overload requiring chelation therapy. Standard-of-care SCD therapies through 2021 included hydroxyurea (1998), L-glutamine/Endari (Emmaus, 2017), crizanlizumab/Adakveo (Novartis, 2019, withdrawn EU 2023), and voxelotor/Oxbryta (Global Blood Therapeutics/Pfizer, 2019, voluntarily withdrawn September 2024). The only curative approach was allogeneic hematopoietic stem cell transplantation, limited by donor availability and graft-versus-host disease. CTX001 (later exagamglogene autotemcel/Casgevy) is an autologous CRISPR/Cas9 ex vivo edited hematopoietic stem cell therapy that disrupts the BCL11A erythroid enhancer to reactivate fetal hemoglobin (HbF). FDA approved Casgevy December 2023 for SCD and January 2024 for TDT — the first CRISPR-edited therapy approved in the US.
At deal date (April 2021), the SCD/TDT competitive landscape was rapidly evolving. Direct CTX001 competitors in gene therapy/gene editing included bluebird bio's lovo-cel (LentiGlobin BB305 lentiviral beta-globin gene addition for SCD, BLA filed 2022, approved Lyfgenia December 2023), bluebird's beti-cel (Zynteglo for TDT, approved August 2022), Editas Medicine EDIT-301 (CRISPR Cas12a HBG1/2 promoter, Phase 1/2 RUBY/EdiThal), Beam Therapeutics BEAM-101 (base editing of HBG1/2 promoter, Phase 1/2), Sangamo/Sanofi BIVV003 (zinc finger nuclease BCL11A, discontinued 2021), and Graphite Bio GPH101 (homology-directed repair, paused 2022 due to safety event). Small molecules and biologics: voxelotor/Oxbryta (GBT/Pfizer, $1.4B 2022 sales before September 2024 withdrawal due to mortality signal), crizanlizumab/Adakveo (Novartis), L-glutamine/Endari (Emmaus), hydroxyurea generics. Pyruvate kinase activators (mitapivat/Pyrukynd from Agios, mitapivat in SCD/thalassemia Phase 3 ENERGIZE, etavopivat/Forma now Novo Nordisk Phase 2/3) target a metabolic mechanism. Vertex consolidated economics from 50/50 to 60/40 (Vertex/CRISPR) by paying CRISPR $900M upfront plus $200M at first regulatory approval — paid Q4 2023 upon Casgevy SCD approval.
| Deal | Year | Value | Outcome |
|---|---|---|---|
| Vertex Pharmaceuticals Incorporated / CRISPR Therapeutics AG (this deal) | 2021 | $1.1B | — |
| 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 / CRISPR Therapeutics AG | 2015 | $2.6B | 85 |
| Vertex Pharmaceuticals Incorporated / Alpine Immune Sciences | 2024 | $4.9B | 78 |
| Vertex Pharmaceuticals Incorporated / Crinetics Pharmaceuticals, Inc. | 2026 | $10.0B | 68 |
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