公司技术平台 - 开发新型小分子药物类别 能够精准调控单个基因表达水平 通过识别DNA序列并招募转录机制实现基因表达上调和下调[1] 疾病治疗领域 - 专注单基因遗传病治疗 针对已知单一基因根源的疾病[2] - 主要研究项目包括弗里德赖希共济失调 Fuchs角膜内皮营养不良 强直性肌营养不良症以及亨廷顿舞蹈症[2] 具体疾病机制 - 弗里德赖希共济失调的致病机制为frataxin基因表达水平过低[3]

Design Therapeutics (DSGN) 2025 Conference September 04, 2025 10:55 AM ET Speaker0All right, we're ready to get started. Welcome everyone. I'm Josh Schimmer from the Cantor Biotech Equity Research Team and pleased to introduce from Design Therapeutics, have Prateek Shah, Chief Executive Officer. We're going back to some GeneTACs. So, Prateek, thanks so much for joining.Why don't you frame Design Therapeutics and give us a quick overview of how the GeneTAC platform works?Speaker1What makes design unique and ...