北海康成-B(01228.HK):與UMass Chan醫學院合作首項研究顯示——新型基因治療有望臨牀應用於SMA
格隆匯5月17日丨北海康成-B(01228.HK)發佈公吿,2022年5月17日,公司於今日在華盛頓特區舉行的第25屆ASGCT年會上,展示與UMass Chan醫學院Horae基因治療中心開展罕見病基因治療研究合作的初步數據。
高光坪實驗室的Jun Xie博士團隊經研究得出結論:與基準載體scAAV9-CMVen/ CB-hSMN1,從內源性hSMN1啟動子表達co-hSMN1的新型第二代scAAV9基因治療在脊髓性肌萎縮症(SMA)小鼠模型中顯示了更強的效力、有效性和安全性。此基準載體和目前美國獲批用於治療SMA基因療法的載體類似。
北海康成全球研究副總裁、本研究作者朱雲祥博士表示,“我們的新型第二代基因治療載體與基準載體的不同之處在於內源性SMN1啟動子控制下的SMN1密碼子基因工程優化,它能夠實現高效且受調控的跨組織基因表達,並有可能改善療效和安全性,同時劑量低於目前在患者在臨牀使用的情況。這將鼓勵我們繼續開發第二代療法,作為治療SMA的潛在最佳基因治療手段。”
Li Weibo罕見病研究所聯席所長、Horae基因治療中心與病毒載體中心主任, UMass Chan醫學院微生物與生理系統教授、Penelope Booth Rockwell生物醫學研究教授,本研究主要作者高光坪博士表示,“我們目前尋求開發的針對SMA的新一代基因療法,得益於自十多年前首個基因療法被開發以來,該領域所取得的進步。”高光坪博士亦為ASGCT前任主席。
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