Silence Therapeutics, a biotechnology company focused on precision-engineered medicines for disease silencing, has announced the publication of its human genomic and preclinical data in the latest issue of Blood, the medical journal of the American Society of Hematology (ASH). The study links iron regulation to polycythemia vera (PV), demonstrating the therapeutic rationale for SLN124, Silence’s clinical candidate for treating PV and other hematological disorders.

The study identified links between PV and variations of the iron-regulating gene HFE in a genome-wide association study (GWAS) of 440 PV cases and over 400,000 healthy controls. In a mouse model of PV, the research also showed that hepcidin, a master regulator of iron availability whose expression is influenced by HFE, governs the red blood cell phenotype in PV.

“PV involves the over-production of red blood cells, leading to a range of adverse symptoms and a high risk of life-threatening cardiovascular events,” said Dr. Ute Schaeper, Drug Discovery Project Leader at Silence and co-author on the paper. “The results we reported in Blood provide a genetic and biological rationale for treating PV with SLN124 by raising hepcidin to control systemic iron levels, thus reducing the red blood cell count. This therapeutic approach could potentially prevent the need for patients to undergo periodic blood withdrawals to treat their disease and reduce their risk of cardiovascular events without the fluid shifts and severe iron deficiency associated with phlebotomies.”

SLN124, an siRNA (short interfering RNA) targeting TMPRSS6, is being studied in the SANRECO phase 1/2 study in adults with PV. It is also being studied in other hematological disorders such as beta-thalassemia. SLN124 works by silencing TMPRSS6, a gene that negatively regulates hepcidin expression, increasing production of hepcidin in the liver.

Silence Therapeutics’ proprietary mRNAi GOLD platform utilizes RNA interference to inhibit the expression of specific target genes implicated in the pathology of diseases with high unmet need. Its wholly-owned product candidates include zerlasiran (SLN360) designed to address the high and prevalent unmet medical need in reducing cardiovascular risk in people born with high levels of lipoprotein, and SLN124 designed to treat hematological disorders. The company also has ongoing research and development collaborations with AstraZeneca, Mallinckrodt Pharmaceuticals, and Hansoh Pharma, among others.

The study’s publication shows a significant breakthrough in the treatment of Polycythemia vera, where current therapies often require patients to undergo periodic blood withdrawals, leading to severe iron deficiency and fluid shifts associated with phlebotomies. This genetic and biological rationale for treating PV with SLN124 provides hope for patients, clinicians, and researchers for a more effective and less invasive treatment option in the future.

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By Jane Lee

Jane Lee is a technology journalist who specializes in writing about the latest innovations and trends in the tech industry. She has covered topics such as artificial intelligence, cybersecurity, and cryptocurrency, and has interviewed some of the most influential figures in the tech world. Jane's engaging writing style and her ability to explain technical concepts in simple terms make her a favorite among tech enthusiasts.

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