北海康成(01228.HK):脊髓性肌肉萎縮症基因療法數據顯示具持久性療效
北海康成(01228.HK)公布,與麻省州立大學醫學院研究協議開發的脊髓性肌肉萎縮症基因療法(CAN203)新型第二代基因療法,通過側腦室內注射改善小鼠壽命和運動功能,數據將於美國第26屆ASGCT年會上公布,結果顯示與基準療法相比,二代基因療法在注射一年後仍保持高水平的SMN蛋白表達,證明療法具持久性療效。
北海康成副總裁兼基因療法研究負責人Jason West表示,在動物模型中使用CAN203進行低劑量ICV輸注產生更具靶向性的SMN蛋白表達分佈,使其有可能成為一種更安全、 更有效的基因療法,並有潛力在直接輸送到中樞神經的情況下用於各年齡段的SMA患者,很高興將CAN203從概念驗證非臨床研究推進到IND授權研究的階段。
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