FDA approves breakthrough gene therapy for a rare immune disorder
美國食品藥物管理局核准一項針對罕見免疫疾病的突破性基因療法
In March 2026, the U.S.
2026年3月，美國食品藥物管理局（FDA）授予Kresladi加速批准，達成了一項歷史性里程碑。
Food and Drug Administration (FDA) reached a historic milestone by granting accelerated approval to Kresladi, a groundbreaking gene therapy designed for children with severe leukocyte adhesion deficiency type I (LAD-I).
這是一種開創性的基因療法，專為患有嚴重第一型白血球黏附缺乏症（LAD-I）的兒童所設計。
This ultra-rare genetic disorder, caused by mutations in the ITGB2 gene, prevents white blood cells from fighting infections, often proving fatal in early childhood.
這種極為罕見的遺傳性疾病，係因ITGB2基因突變所致，會導致白血球無法對抗感染，且常在幼兒時期便致命。
Kresladi represents a major medical breakthrough as the first-ever approved treatment for this condition.
Kresladi是針對此病症首個獲批的療法，堪稱重大醫學突破。
Developed by Rocket Pharmaceuticals, the therapy is autologous, meaning it uses a patient’s own modified stem cells to restore essential immune function.
該療法由Rocket Pharmaceuticals公司開發，屬於自體（ㄗˋㄊㄧˇ）療法，意即利用病患自身的修飾幹細胞來恢復必要的免疫功能。
By reintroducing healthy copies of the ITGB2 gene, the treatment helps the body effectively clear dangerous bacteria and fungi.
透過重新導入健康的ITGB2基因副本，該療法能協助身體有效清除危險的細菌與真菌。
Donald Kohn at UCLA, demonstrated that patients experienced fewer severe infections and sustained biomarker improvements over time.
過去，病患只能依賴來自配對捐贈者的高風險幹細胞移植，但並非總能找到合適對象。
In recognition of this effort to address a critical pediatric need, the FDA also awarded the company a Rare Pediatric Disease Priority Review Voucher.
