Table 2.

Novel Therapeutics in β-Thalassemia

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

NTDT non-transfusion-dependent thalassemia

TDT transfusion-dependent thalassemia