Drug Pipeline

28 drugs associated with Beta-thalassemia

DrugPhaseSponsor
Sodium 2,2 dimethylbutyrate
Phase 2Boston University
SPD602 (FBS0701, SSP-004184)
Phase 2Shire
deferasirox

Two molecules of deferasirox are capable of binding to 1 atom of iron. Deferasirox works in treating iron toxicity by binding trivalent (ferric) iron (for which it has a strong affinity), forming a stable complex which is eliminated via the kidneys.

Phase 3Novartis
Amlodipine

**Mechanism of action on blood pressure** Amlodipine is considered a peripheral arterial vasodilator that exerts its action directly on vascular smooth muscle to lead to a reduction in peripheral vascular resistance, causing a decrease in blood pressure. Amlodipine is a dihydropyridine calcium antagonist (calcium ion antagonist or slow-channel blocker) that inhibits the influx of calcium ions into both vascular smooth muscle and cardiac muscle. Experimental studies imply that amlodipine binds t

unknownTanta University
Curcuminoids and alpha-tocopherol
unknownMahidol University
Luspatercept

Luspatercept-aamt is a recombinant fusion protein that binds several endogenous TGF-β superfamily ligands, thereby diminishing Smad2/3 signaling. In models of β-thalassemia and MDS, luspatercept-aamt decreased abnormally elevated Smad2/3 signaling and improved hematology parameters associated with ineffective erythropoiesis in mice. Luspatercept-aamt promoted erythroid maturation through differentiation and increasing the percentage of late-stage erythroid precursors (normoblasts) in the bone ma

unknownBristol-Myers Squibb
Spirulina
unknownTanta University
γ-globin reactivated autologous hematopoietic stem cells

Somatic cell supplemental therapy

unknownBioray Laboratories