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Silence Therapeutics' Thalassemia Hopeful Shows Favorable Safety Profile In Early Stage Study

Silence Therapeutics' Thalassemia Hopeful Shows Favorable Safety Profile In Early Stage Study

Silent Treatetics的地中海贫血有望在早期研究中显示出良好的安全性
Benzinga Real-time News ·  2022/09/29 11:26
  • Silence Therapeutics plc (NASDAQ:SLN) announced preliminary results from the single dose component of the GEMINI II phase 1 study of SLN124 in 24 adults with non-transfusion-dependent thalassemia.
  • The primary objective of the single dose arm was to evaluate the safety and tolerability of SLN124 subcutaneous dosing (1.0, 3.0, and 6.0 mg/kg) in alpha/beta-thalassemia patients.
  • Following a single dose, there were no serious adverse events, severe treatment-emergent adverse events (TEAEs) that were SLN124 related, or TEAEs leading to withdrawal.
  • No dose-limiting toxicities or drug-related liver injuries were observed.
  • Pharmacokinetic (PK) parameters and pharmacodynamic (PD) biomarkers of iron metabolism are being evaluated in the study's ongoing multiple dose arm, which is anticipated to read out next year.
  • 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.
  • Price Action: SLN shares are trading at $10.00 on the last check Thursday.
  • 沉默治疗公司纳斯达克公司(Sequoia Capital：SLN)宣布了对24名成年非输血依赖型地中海贫血患者进行的双子座II第一阶段SLN124单剂试验的初步结果。
  • 单剂ARM的主要目标是评估SLN124皮下给药(1.0、3.0和6.0 mg/kg)对阿尔法/贝塔地中海贫血患者的安全性和耐受性。
  • 在单次服药后，没有发生严重不良事件、与SLN124相关的严重治疗紧急不良事件(TEAE)或导致停药的TEAE。
  • 没有观察到剂量限制性毒性或药物相关的肝损伤。
  • 铁代谢的药代动力学(PK)参数和药效学(PD)生物标志物正在进行研究的多剂量ARM中进行评估，预计将于明年读出。
  • SLN124是一种基因沉默疗法--一种旨在暂时阻断特定基因信息的疗法，否则会引发不必要的影响。在这种情况下，SLN124的目标是暂时“沉默”TMPRSS6，这是一种基因，可以防止肝脏产生一种控制体内铁水平的特定荷尔蒙--海普西丁。
  • 价格行动：在周四的最后一张支票上，SLN的股票交易价格为10.00美元。
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