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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