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SLN124

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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.

Learn More About Thalassemia
Learn More About
Polycythemia Vera

In May 2021, we completed the GEMINI phase 1 study of SLN124 in healthy volunteers. We are currently evaluating SLN124 in the GEMINI II phase 1 study in adults with thalassemia and the SANRECO phase 1/2 study in adults with polycythemia vera (PV).

The U.S. FDA has granted SLN124 Fast Track and orphan drug designations for PV as well as orphan drug and rare pediatric disease designations for beta-thalassemia. The European Medical Agency has also granted SLN124 orphan drug and rare pediatric disease designations for beta-thalassemia.

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Learn More About the
SANRECO Phase 1/2 PV Study

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Learn More About the
GEMINI II Patient Study

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Learn More About the
GEMINI Healthy Volunteer Study