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信念医药杜氏肌营养不良症基因治疗药物BBM-D101注射液IND获NMPA受理
BBM-D101注射液是信念医药拥有自主知识产权的腺相关病毒(AAV)基因治疗药物。通过单次静脉输注,基于工程化AAV载体,将优化的基因表达盒递送到全身肌肉,以期实现“一次给药、长期有效”的治疗DMD。BBM-D101注射液生产则运用了公司自主开发的500L无血清悬浮培养工艺,符合《药品生产质量管理规范》(GMP)要求。
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