Ultragenyx Pharmaceutical（纳斯达克代码：RARE）近日宣布，美国食品药品监督管理局（FDA）已正式受理其针对UX111 AAV基因疗法的生物制品许可申请（BLA）重新提交。该疗法旨在治疗A型圣菲利波综合征（又称黏多糖贮积症IIIA型，MPS IIIA）。
此次BLA重新提交标志着公司在推进该罕见病治疗领域的重要进展。UX111作为一种创新基因疗法，通过腺相关病毒载体递送功能性基因，有望为MPS IIIA患者提供新的治疗选择。
FDA的受理决定将启动相关审评流程，后续进展备受市场关注。
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