Beta-Thalassemia

Hematology
8
Pipeline Programs
4
Companies
6
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
2
5
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

VP
4 programs
1
3
CTX001Phase 31 trial
CTX001Phase 31 trial
CTX001Phase 31 trial
CTX001Phase 2/31 trial
Active Trials
NCT03655678Completed59Est. Nov 2025
NCT05477563Recruiting26Est. Jun 2027
NCT04208529Enrolling By Invitation160Est. Sep 2039
+1 more trials
CRISPR Therapeutics
3 programs
1
2
CTX001Phase 3
CTX001Phase 3
CTX001Phase 2/3
Sanofi
SanofiPARIS, France
2 programs
1
DeferitrinPhase 1/21 trial
MozobilN/A1 trial
Active Trials
NCT01206075Completed20Est. Dec 2014
NCT00069862Completed25Est. Nov 2005

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Vertex PharmaceuticalsCTX001
Vertex PharmaceuticalsCTX001
Vertex PharmaceuticalsCTX001
Vertex PharmaceuticalsCTX001
SanofiDeferitrin
SanofiMozobil

Clinical Trials (6)

Total enrollment: 306 patients across 6 trials

NCT05477563Vertex PharmaceuticalsCTX001

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Start: Aug 2022Est. completion: Jun 202726 patients
Phase 3Recruiting
NCT05356195Vertex PharmaceuticalsCTX001

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Start: May 2022Est. completion: May 202616 patients
Phase 3Active Not Recruiting
NCT04208529Vertex PharmaceuticalsCTX001

A Long-term Follow-up Study in Participants Who Received CTX001

Start: Jan 2021Est. completion: Sep 2039160 patients
Phase 3Enrolling By Invitation
NCT03655678Vertex PharmaceuticalsCTX001

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Start: Sep 2018Est. completion: Nov 202559 patients
Phase 2/3Completed
NCT00069862SanofiDeferitrin

Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

Start: Sep 2003Est. completion: Nov 200525 patients
Phase 1/2Completed
NCT01206075SanofiMozobil

Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major

Start: Oct 2010Est. completion: Dec 201420 patients
N/ACompleted

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

5 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 306 patients
4 companies competing in this space

Related Indications

Anemia(92)Myelofibrosis(21)Polycythemia Vera(16)Primary Immune Thrombocytopenia(15)Primary Myelofibrosis(14)Immune Thrombocytopenia(12)Thrombocytopenia(11)Aplastic Anemia(7)Anemia of Chronic Kidney Disease(7)Iron Deficiency Anemia(6)