Table 1 Novel therapeutics in Sickle Cell Disease
From: Erythropoiesis: insights into pathophysiology and treatments in 2017
| a. Gene therapy | ||||||
| Bluebird Bio | BB305 lentiviral vector (betibeglogene darolentivec) | Anti-sickling β-globin |
- Severe SCD - age ≥ 18 years | IV | NCT02140554 |
Open - Phase 1/2 |
| Children’s Hospital Medical Center, Cincinnati | Gamma globin lentiviral vector | Anti-sickling γ-globin |
- Severe SCD - age 18–35 | IV | NCT02186418 |
Open - Phase 1/2 |
| b. Small molecule targets | ||||||
| Boston University | SIRT1 | HbF induction |
Early stage of development - pre-clinical | |||
| The Cleveland Clinic | Decitabine and tetrahydrouridine | HbF induction |
- age ≥ 18 years - HbSS, HbSβ°, HbSβ+, HbSC | Oral | NCT01685515 |
Completed - Phase 1 |
| Celgene | Pomalidomide | HbF induction |
- age 18–60 - HbSS, HbSβ° | Oral | NCT01522547 |
Completed - Phase 1 |
| Novartis Pharmaceuticals | Panobinostat | HbF induction |
- age ≥ 18 yers - HbSS, HbSβ° | Oral | NCT01245179 |
Open - Phase 1 |
| Dana Farber Cancer Institute | Vorinostat (Zolinza) | HbF induction |
- age 18–60 - HbSS, HbSβ° | Oral | NCT01000155 |
Discontinued - Phase 1/2 |