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Table 2 Novel Therapeutics in β-Thalassemia

From: Erythropoiesis: insights into pathophysiology and treatments in 2017

Company Drug/Target Mechanism Eligibility Route Clintrials.gov Status
a. Gene therapy
 Bluebird Bio BB305 lentiviral vector (betibeglogene darolentivec) Improved erythropoiesis - Transfusion-dependent β-Thalassemia
-age 12–35
IV NCT01745120 Active, not recruiting
- Phase 1/2
 IRCCS San Raffaele GLOBE lentiviral vector Improved erythropoiesis - Transfusion-dependent β-Thalassemia
-age ≥ 3 and < 65
IV NCT02453477 Open
- Phase 1/2
b. Small molecule targets
 New England Research Institutes Decitabine HbF induction - age ≥ 18
- TD β Thalassemia and HbEβ-Thalassemia
Subcutaneously NCT00661726 Completed
- Phase 2
 Medical College Kolkata Decitabine HbF induction - age ≥ 18
- TDT and NDTD HbEβ-Thalassemia
Subcutaneously Completed
- ASH 2017
 Novartis Pharmaceuticals INC424 (Ruxolitinib) Jak 1/2 inhibitor - age ≥ 18
- TDT β- Thalassemia
- Splenomegaly
- iron chelation × 4 weeks
Oral NCT02049450 Completed
- Phase 2a
Acceleron ACE-536 (Luspatercept) Ligand trap TBG beta superfamily - age ≥ 18
- TD and NTDT β-Thalassemia
Subcutaneously NCT02268409 Active, not recruiting
- Phase 2
Celgene ACE-011 (Sotatercept) Ligand trap TBG beta superfamily - age ≥ 18
- TD and NTDT β-Thalassemia
Subcutaneously NCT01571635 Active, not recruiting
- Phase 2a
  1. NTDT non-transfusion-dependent thalassemia
  2. TDT transfusion-dependent thalassemia