Gene Therapy Platform for Rare Diseases

TRND计划启动了与生物技术和学术团体的一系列合作，这些团体被战略地选择作为首批试点项目。总体目标是使TRND能够建立一个基因治疗翻译工具箱。TRND正在开发的新技术包括扩大基因载体制造规模，并在正确的时间和剂量将转基因输送到正确的组织。这些技术，以及实现基因治疗监管批准的最佳实践，将有助于提高基因治疗的开发速度，并总体上降低成本。试点项目包括治疗Duchenne肌营养不良症、庞贝病和芳香族L氨基酸脱羧酶缺乏症的临床前开发。

The TRND Program initiated a suite of collaborations with biotech and academic groups that were strategically selected to serve as inaugural pilot projects. The overall goal is to enable TRND to build a gene therapy translation toolbox. New technologies to scale up gene vector manufacturing and to deliver the transgene to the right tissue at the right time and dosage are among those being developed at TRND. These technologies, along with best practices to achieve regulatory approval of gene therapy, will help improve the speed of development and reduce costs for gene therapy in general. The pilot projects include preclinical development of therapies for Duchenne muscular dystrophy, Pompe disease, and aromatic L-amino acid decarboxylase (AADC) deficiency.