Gene Therapy Platform for Rare Diseases

TRND计划发起了一系列与生物技术和学术团体的合作，这些团体被战略性地选为首批试点项目。总体目标是使TRND能够建立一个基因治疗翻译工具箱。扩大基因载体制造和在正确的时间和剂量将转基因输送到正确的组织的新技术是TRND正在开发的技术之一。这些技术，沿着最佳实践，以实现基因治疗的监管批准，将有助于提高发展速度和降低成本的基因治疗一般。试点项目包括杜氏肌营养不良症、庞贝氏症和芳香族L-氨基酸脱羧酶（AADC）缺乏症的临床前治疗开发。

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.