Silence Therapeutics' siRNA Candidate Reduces Plasma Iron Levels, Early-Stage Study Shows
- Silence Therapeutics plc (NASDAQ: SLN) has announced topline data from GEMINI Phase 1 study evaluating SLN124 for iron-loading anemia conditions, thalassemia, and myelodysplastic syndrome (MDS).
- SLN124 is a short interfering ribonucleic acid (siRNA) that targets TMPRSS6.
- The single-ascending dose study evaluated the safety and tolerability of SLN124 in 24 healthy volunteers.
- Pharmacokinetic parameters and pharmacodynamic biomarkers of iron metabolism were also measured to assess the reduction in iron.
- Initial data from the study showed all doses of SLN124 were generally well-tolerated with no serious or severe treatment-emergent adverse events.
- The adverse events did not appear to be dose-dependent, and the majority were mild, including transient injection site reactions, which resolved without intervention.
- Up to a four-fold increase in average hepcidin and a 50% reduction in plasma iron levels were also observed after a single dose of SLN124.
- Hepcidin is a protein that acts as a key regulator of the entry of iron into the circulation in mammals.
- Effects on hepcidin and iron appeared to be dose-dependent and were still observed at the end of the 8-week study at all dose levels.
- Price Action: SLN shares closed at $25.65 on Tuesday.
© 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Posted-In: Anemia Briefs Phase 1 TrialBiotech News Health Care Small Cap General
