New therapeutic approaches for thalassemia

 
Maria Domenica Cappellini, Olivier Hermine, Antonio Piga, et al.
 
The authors of the abstract conclude: 
Although response rates were lower in pts with the most severe disease (β00), clinically meaningful reductions in transfusion burden were observed across genotypes. 
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John B. Porter, Alexis A. Thompson, Mark C. Walters, et al.
 
The authors of the abstract conclude: 
Following treatment with betibeglogene autotemcel (beti-cel; LentiGlobin for β-thalassemia) gene therapy, 9/10 patients with TDT and non-β00 genotypes achieved transfusion independence. These patients showed improvements in erythropoiesis, suggesting that the the bone marrow is in process of normalizing. The treatment regimen comprised of conditioning and beti-cel infusion had a tolerability profile consistent with the known effects of busulfan myeloablation.
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Kevin HM Kuo, D Mark Layton, Ashutosh Lal, et al.
 
The authors of the abstract conclude: 
These data establish proof of concept that activation of wild-type PK-R by mitapivat has potential clinical benefit in thalassemia, and support its further investigation as an oral treatment for pts with β- and α-thalassemia. This study is also the first drug trial aimed at treating anemia in α-thalassemia.
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Ashutosh Lal, Ersi Voskaridou, P Flevari, et al.
 
The authors of the abstract conclude: 
Though preliminary, these results demonstrate the pharmacodynamic activity of PTG-300 in reducing TSAT and serum iron in subjects with TD β-thalassemia. It is too early to determine whether PTG-300 has clinically meaningful activity in subjects with transfusion-dependent β-thalassemia.
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Maria Rosa Lidonnici, Giulia Chianella, Francesca Tiboni, et al.
 
The authors of the abstract conclude: 
Overall, these data indicate that Bthal HSCs are more cycling cells which egress from the quiescent state probably towards an erythroid differentiation, probably in response to a chronic BM stimulation. On the other hand, some evidence support our hypothesis of an ‘erythroid branching’ already present in the HSC pool, exacerbated by the pathophysiology of the disease.