In a first, FDA approves a gene therapy from a nonprofit
FDA首次批准非营利组织开发的基因疗法

The FDA on Tuesday approved a gene therapy for a rare immune disease called Wiskott-Aldrich syndrome, for which patients have little options besides a bone marrow transplant.
美国食品药品监督管理局(FDA)周二批准了一种用于治疗罕见免疫疾病——Wiskott–Aldrich综合征——的基因疗法。除骨髓移植外，患者几乎别无选择。