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 |