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成都先導(688222.SH):目前成都先導肝內靶向的GalNAc在分子水平和細胞水平已經驗證過
格隆匯 11-02 09:52

格隆匯11月2日丨成都先導(688222.SH)11月1日接受特定對象調研時表示,目前成都先導肝內靶向的GalNAc在分子水平和細胞水平已經驗證過,目前正在進行體內驗證;基於DEL篩選平台識別TfR1小分子配體介導的肝外遞送系統以及潛在CNS系統遞送的研發也在同步推進中。目前大部分小核酸藥物的靶點都是在肝臟,不管是脂質體納米顆粒遞送,還是共軛偶聯遞送,肝細胞會高度表達ASGPR受體,共軛偶聯後很容易富集核酸藥到肝臟並且進入肝細胞內部,但是隻能解決肝臟的問題,實際上在人體內還有很多其他的受體配體等,TfR1配體也許可以做到肝外遞送。

成都先導在遞送領域有獨特的研發優勢。過去的遞送方式主要針對器官和組織,我們認為下一代的遞送技術更針對特定細胞的選擇性遞送(類似GalNAc遞送,加一個配體),全球來看,這種技術就是將核酸藥物直接和細胞進行接觸,不需要LNP仍然完成遞送以達到治療效果,我們的DEL技術在新型分子的發現上有明顯的優勢,有可能找到全新的遞送分子。

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