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Alnylam Presents Preclinical Data on Beta-Thalassemia, Erythropoiesis Efforts


Alnylam Pharmaceuticals this week released new preclinical data from programs in beta-thalassemia and erythropoiesis — indications from which the company could select its newest pipeline candidate.

Alnylam, which presented the data at the American Society of Hematology annual meeting, said that it and collaborators showed that the systemic administration of a lipid nanoparticle-formulated siRNA targeting the gene transmembrane protease, serine 6, triggered an increase in hepcidin mRNA levels in mouse models of iron overload in beta-thalassemia.

As a result, the mice experienced significant decreases in serum iron concentration and transferrin saturation.

“Moreover, Tmprss6 silencing with a single dose of the RNAi therapeutic was associated with a dramatic normalization of multiple hematological parameters including hemoglobin, normalization of splenic iron levels and tissue histology, and reductions in splenomegaly,” Alnylam said. “These findings suggest that iron restriction via Tmprss6 silencing can correct the disease phenotype of beta-thalassemia intermedia in a pre-clinical model.”

Company researchers also reported that systemically delivered lipid nanoparticles loaded with siRNAs against liver-expressed egl-nine homolog prolyl hydroxlase genes could induce hepatic erythropoietin mRNA activation, which led to increased serum erythropoietin levels and stimulation of red blood cell production in animal models.

“Following a single dose, increases in serum EPO and hematocrit were durable for approximately two weeks and one month, respectively,” Alnylam said, adding that the treatment corrected anemia in both renal failure and inflammatory anemia disease models.

In a statement, Alnylam CMO Akshay Vaishnaw said that the company remains on track to announce a new drug candidate by year end.

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