CTX001

Phase 3Active
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76
Hype Score

Development Stage

โœ“
Pre-clinical
โœ“
Phase 1
โœ“
Phase 2
4
Phase 3
5
Approved
Indication / Disease

Beta-Thalassemia

Conditions

Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies

Trial Timeline

May 3, 2022 โ†’ May 31, 2026

About CTX001

CTX001 is a phase 3 stage product being developed by Vertex Pharmaceuticals for Beta-Thalassemia. The current trial status is active. This product is registered under clinical trial identifier NCT05356195. Target conditions include Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn.

Hype Score Breakdown

Clinical
27
Activity
18
Company
9
Novelty
9
Community
10

Clinical Trials (6)

NCT IDPhaseStatus
NCT05477563Phase 3Recruiting
NCT05356195Phase 3Active
NCT05329649Phase 3Active
NCT04208529Phase 3Recruiting
NCT03745287Phase 2/3Completed
NCT03655678Phase 2/3Completed

Competing Products

18 competing products in Beta-Thalassemia

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ACE-536MerckPhase 2
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DeferasiroxNovartisApproved
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Zoledronic acid + PlaceboNovartisPhase 2/3
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deferasiroxNovartisPhase 3
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ICL670 + deferoxamineNovartisPhase 3
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DeferasiroxNovartisPhase 2
52
DeferasiroxNovartisPhase 2
52
ICL670NovartisApproved
85
BitopertinRochePhase 2
52
Luspatercept + PlaceboBristol Myers SquibbPhase 2
51
CTX001Vertex PharmaceuticalsPhase 3
76
CTX001Vertex PharmaceuticalsPhase 2/3
64
CTX001Vertex PharmaceuticalsPhase 3
76
Mozobil + Mozobil + MozobilSanofiPre-clinical
22
Deferitrin (GT56-252) + desferoxamine (DFO)SanofiPhase 1/2
40
REGN7999 + PlaceboRegeneron PharmaceuticalsPhase 2
51
VIT-2763 60 mg QD + VIT-2763 60 mg BID + VIT-2763 120 mg BID + PlaceboLabCorpPhase 2
49
VIT-2763 once a day (QD) + VIT-2763 twice a day (BID) + PlaceboLabCorpPhase 2
49