On June 7, 2021 Silence Therapeutics plc, AIM:SLN and Nasdaq: SLN ("Silence" or "the Company"), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, reported that it will present data showing the potential for SLN124 to address a range of hematological diseases by targeting the liver-expressed gene TMPRSS6 during two poster sessions at the European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress to be held on June 9-17, 2021 (Press release, Silence Therapeutics, JUN 7, 2021, View Source [SID1234583654]).

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The presentations include a preclinical safety assessment of SLN124 which will show data that are consistent with recent positive topline clinical data from the GEMINI phase 1 study of SLN124 in healthy volunteers announced last month (press release linked here). SLN124 is being evaluated in the ongoing GEMINI II phase 1 study in patients with thalassemia and myelodysplastic syndrome. A second poster will present encouraging preclinical evidence that using siRNA to target TMPRSS6 could be a viable therapeutic avenue for treatment of the rare blood disorder, polycythaemia vera (PV).

E-posters will be available for registered attendees through the EHA (Free EHA Whitepaper) Virtual Congress platform starting Friday, June 11, 09:00 CEST.

Details on the presentations are as follows:

Title: Non-clinical safety of SLN124, a GalNAc conjugated 19-mer double stranded siRNA targeting TMPRSS6 facilitating evaluation in clinical studies

Abstract #: EP846 (link here)

Session Topic: Iron metabolism, deficiency and overload

Title: Anti-TMPRSS6 RNAi Therapy as a Novel Treatment Option for Polycythaemia Vera

Abstract #: EP1057 (link here)

Session Topic: Myeloproliferative neoplasms – Biology & Translational Research

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, it is hoped that iron levels in the blood will decrease, which could in turn allow more healthy red blood cells to be produced, thereby improving anemia.

​In preclinical studies, SLN124 showed a strong safety profile and positive effects on improving levels of red blood cells and reducing harmful iron levels. Data from the recently completed GEMINI phase 1 study of SLN124 in healthy volunteers demonstrated SLN124 was safe and effective in reducing plasma iron levels and had a long duration of action. These data support the ongoing GEMINI II phase 1 study of SLN124 in people with thalassemia or myelodysplastic syndrome (MDS), whose bodies produce fewer healthy red blood cells than normal and who can store too much iron in their bodies. For more information on the GEMINI II study, please click here.