Investing.com -- Precision BioSciences (NASDAQ:DTIL)已获得美国食品药品监督管理局(FDA)对其靶向杜氏肌营养不良症的核酸酶治疗的孤儿药资格认定。
该认定授予了该公司的腺相关病毒9型递送的DMD19-20L.431和DMD35-36L.457核酸酶，这些核酸酶正在开发用于治疗杜氏肌营养不良症。
孤儿药资格认定授予用于治疗、诊断或预防在美国影响不足20万人的罕见疾病的药物和生物制品。这一地位为药物开发商提供了某些优势，包括合格临床试验的税收抵免和潜在的市场独占权。
杜氏肌营养不良症是一种罕见的遗传性疾病，特征为肌肉逐渐退化和无力，这是由于一种称为肌营养不良蛋白的蛋白质发生改变，该蛋白质有助于保持肌肉细胞的完整性。
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