基因疗法治疗罕见病的希望与陷阱
由于最近的监管和临床挫折使人们对基因疗法的前景产生了疑问，基因疗法的现状面临着许多不确定性。迄今为止，美国食品及药物管理局只批准了两种用于罕见病的基因疗法，因此开发这些药物的道路依然艰难。This presentation exploreed how the biopharma industry can overcome development challenges in the … Continued
Tag: 细胞和基因治疗
Commercial and Regulatory Success in Accelerated Gene Therapy Development
Thanks to regulatory frameworks and driven by high unmet patient needs, the pace for clinical development of gene therapies (GT) is often greatly accelerated. This presents GT innovators with the opportunity to reach their patients faster, but it also raises the bar for critical decision points such as around trial design, endpoint selection and population … Continued
为患者提供基因疗法
This blog discusses the challenges that drug developers face in getting patients access to cell and gene therapies as well as some potential solutions.
基因疗法临床试验设计中的早期价值评估
利用基因治疗虚拟双胞胎平台优化剂量
This blog explains how using the Gene Therapy Virtual Twin™ Platform can help optimize dosing for patients.
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