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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