美國批准首個基因編輯療法Casgevy醫鐮狀細胞疾病 費用220萬美元
美國食品藥物管理局(FDA)周五批准該國首個基因編輯療法Casgevy,代表著一項重大的科學進步,療法適用於12歲及以上鐮狀細胞疾病患者。
鐮狀細胞疾病是一種遺傳性疾病,會影響紅血球,導致紅血球變得僵硬和黏稠,並破壞向組織輸送氧氣。根據美國疾病管制與預防中心的估計,美國約有10萬人患有該疾病,其中黑人受到的影響尤其嚴重。
Casgevy由Vertex Pharmaceuticals(VRTX.US)和CRISPR(CRSP.US)共同開發,利用諾貝爾獎得主技術CRISPR編輯人的基因以治療疾病。該療法上個月獲得英國監管機構的批准。
Vertex將率先推出該藥物,估計約16,000名鐮狀細胞重症患者將有資格獲得該藥物,費用為220萬美元。
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