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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的地中海貧血有望在早期研究中顯示出良好的安全性
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.
  • 沉默治療公司納斯達克公司(Sequoia Capital：SLN)宣佈了對24名成年非輸血依賴型地中海貧血患者進行的雙子座II第一階段SLN124單劑試驗的初步結果。
  • 單劑ARM的主要目標是評估SLN124皮下給藥(1.0、3.0和6.0 mg/kg)對阿爾法/貝塔地中海貧血患者的安全性和耐受性。
  • 在單次服藥後，沒有發生嚴重不良事件、與SLN124相關的嚴重治療緊急不良事件(TEAE)或導致停藥的TEAE。
  • 沒有觀察到劑量限制性毒性或藥物相關的肝損傷。
  • 鐵代謝的藥代動力學(PK)參數和藥效學(PD)生物標誌物正在進行研究的多劑量ARM中進行評估，預計將於明年讀出。
  • SLN124是一種基因沉默療法--一種旨在暫時阻斷特定基因信息的療法，否則會引發不必要的影響。在這種情況下，SLN124的目標是暫時“沉默”TMPRSS6，這是一種基因，可以防止肝臟產生一種控制體內鐵水平的特定荷爾蒙--海普西丁。
  • 價格行動：在週四的最後一張支票上，SLN的股票交易價格為10.00美元。
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