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Addressing Hematological Diseases

SLN124 is our wholly owned siRNA (short interfering RNA) in development for hematological diseases.

About SLN124

SLN124 is a gene ‘silencing’ therapy – one that is designed to temporarily block a specific gene’s message that would otherwise trigger an unwanted effect. In this case, SLN124 aims to temporarily ‘silence’ TMPRSS6, a gene that prevents the liver from producing a particular hormone that controls iron levels in the body – hepcidin. As hepcidin increases, iron levels in the blood are expected to decrease, which may increase the production of healthy red blood cells, thereby reducing anemia.

In May 2021, we completed the GEMINI phase 1 study of SLN124 in healthy volunteers. Data from the study showed SLN124 was safe and effective in reducing plasma iron levels and had a long duration of action.

We are evaluating SLN124 in the GEMINI II phase 1 study in adults with thalassemia, whose bodies produce fewer healthy red blood cells than normal and who can store too much iron in their bodies. SLN124 has orphan drug designation and rare pediatric disease designation for thalassemia.