Precision BioSciences获FDA孤儿药资格认定用于治疗杜氏肌营养不良症

英为财情
Jul 18, 2025

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...

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