ProQR Receives $9.1M to Develop RNA Therapy for Rett Syndrome, Impacting 350,000 People
ProQR Receives $9.1M to Develop RNA Therapy for Rett Syndrome, Impacting 350,000 People
ProQR Therapeutics NV. (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today announced an expansion of its collaboration with the Rett Syndrome Research Trust (RSRT). Building on the initial $1 million research grant announced in January 2024, the expanded partnership includes an additional $8.1 million in funding from the RSRT, for a total of $9.1 million. The funding will support the advancement of AX-2402 into clinical trials.
ProQR Therapeutics NV.（纳斯达克：PRQR）（ProQR）是一家致力于通过其专有的Axiomer RNA编辑技术平台改变生活的公司，今天宣布扩大与Rett综合症研究信托（RSRT）的合作。在2024年1月宣布的初始100万美元研究资金的基础上，扩展的合作关系还包括来自RSRT的额外810万美元资金，总计910万美元。这笔资金将支持AX-2402进入临床试验的进展。
Rett Syndrome, a rare and devastating neurodevelopmental disorder, affects approximately 350,000 people worldwide, predominantly girls. With no current cures and limited treatment options, Rett Syndrome presents a significant unmet medical need. AX-2402 is being developed for individuals with Rett syndrome who have the R270X mutation in MECP2 gene, and is based on ProQR's proprietary Axiomer RNA editing platform. Axiomer can target many mutations beyond R270X that collectively impact a large segment of the Rett population. Success with AX-2402 sets the foundation for developing RNA editing therapeutics to target the remaining mutations.
Rett综合症是一种罕见且毁灭性的神经发育障碍，全球约有350,000人受其影响，主要是女孩。目前没有治愈方法，治疗选择有限，Rett综合症提出了显著的未满足医疗需求。AX-2402正在为携带MECP2基因中R270X突变的Rett综合症患者开发，基于ProQR的专有Axiomer RNA编辑平台。Axiomer可以针对许多超出R270X的突变，这些突变共同影响了Rett人群的一个大部分。AX-2402的成功为开发RNA编辑治疗药物以针对其余突变奠定了基础。
