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CTX001

Phase 3

Sickle Cell Disease | Monoclonal antibody | Hematology |Vertex Pharmaceuticals Incorporated|Last Updated: Mar 9, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment76
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05329649Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)PHASE3 ACTIVE NOT_RECRUITING 13 — — May 2, 2022May 31, 2026Mar 9, 20267 United States, Germany +2
NCT03745287A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell DiseasePHASE2 COMPLETED 63 — — Nov 27, 2018Jul 7, 2025Aug 11, 202517 United States, Belgium +5
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Study Endpoints
Primary Endpoints
Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12)
Up to 24 Months After CTX001 Infusion
Proportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12)
From 60 days after last RBC transfusion up to 2 years after CTX001 infusion
Proportion of subjects with engraftment (first day of three consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days)
Within 42 days after CTX001 infusion
Time to engraftment
From CTX001 infusion up to 2 years after CTX001 infusion
Frequency and severity of collected adverse events (AEs)
From screening to 2 years after CTX001 infusion
Incidence of transplant-related mortality (TRM) within 100 days after CTX001 infusion
Within 100 days after CTX001 infusion
Incidence of TRM within 1 year after CTX001 infusion
Within 1 year after CTX001 infusion
All-cause mortality
2 years after mobilization
Secondary Endpoints
Proportion of Participants Free from Inpatient Hospitalization for Severe VOCs for at Least 12 Months (HF12)
Up to 24 Months After CTX001 Infusion
Relative Reduction in Annualized Rate of Severe VOCs
From Baseline up to 24 Months After CTX001 Infusion
Duration of Severe VOC Free in Participants who Have Achieved VF12
Up to 24 Months After CTX001 Infusion
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CTX001EXPERIMENTALCTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive single infusion of CTX001 through central venous catheter.
Interventions
NameTypeDescription
CTX001BIOLOGICALAdministered by intravenous infusion following myeloablative conditioning with busulfan.
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Eligibility Criteria
Age Range2 Years — 11 Years
SexALL
Healthy VolunteersNo
Study Sites7

Key Inclusion Criteria: * Diagnosis of severe SCD as defined by: * Documented SCD genotypes * History of at least two severe VOCs events per year for the previous two years prior to enrollment * Hydroxyurea (HU) failure unless HU intolerant * Eligible for autologous stem cell transplant as per inve...

Countries:United StatesGermanyItalyUnited KingdomBelgiumCanadaFrance
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Competitive Landscape -Sickle Cell Disease 28 trials
CompanyTickerTrialsLead PhaseDrugs
Novo Nordisk A/S Sponsored ADR Class BNVO5PHASE3Etavopivat Low dose, Etavopivat, Etavopivat A, Etavopivat B, Etavopivat C
Novartis AG Sponsored ADRNVS4PHASE3Crizanlizumab, OTQ923, ITU512
Sanofi SA Sponsored ADRSNY2PHASE3PCV21, 20vPCV, Rilzabrutinib
Vertex Pharmaceuticals IncorporatedVRTX3PHASE3CTX001
Agios Pharmaceuticals, Inc.AGIO2PHASE2Mitapivat, Tebapivat
Pfizer Inc.PFE1PHASE2Osivelotor
Fulcrum Therapeutics, Inc.FULC2PHASE2Pociredir
Bristol-Myers Squibb CompanyBMY1PHASE1BMS-986470, Famotidine
Beam Therapeutics, Inc.BEAM2PHASE1BEAM-101, Long-Term Follow-up Study of patients who received BEAM-101
Editas Medicine, Inc.EDIT2PHASE1EDIT-301
Disc Medicine, Inc.IRON1PHASE1DISC-3405
Illumina, Inc.ILMN1 — Undisclosed
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05329649primaryCompletionDate: changed
LOWMay 24, 2026NCT05329649studyFirstPostDate: changed