Solid Biosciences Inc. has received Fast Track designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, SGT-501.

Solid Biosciences Inc. 公司的在研基因疗法 SGT-501 获得了美国食品药品监督管理局 (FDA) 的快速通道资格。

CPVT affects approximately 1 in 10,000 people globally. It typically emerges in childhood or adolescence and is often misdiagnosed. The condition is linked to mutations in the RYR2 and CASQ2 genes, leading to abnormal calcium handling in heart cells. This can cause dangerous arrhythmias during physical activity or emotional stress, with symptoms ranging from fainting and seizures to sudden cardiac arrest..

CPVT在全球范围内大约每1万人中就有1人受到影响。它通常在儿童或青少年时期出现，并且经常被误诊。这种疾病与RYR2和CASQ2基因的突变有关，导致心肌细胞内钙处理异常。这可能在体力活动或情绪压力期间引发危险的心律失常，症状从晕厥、癫痫发作到突发性心脏骤停不等。

Currently, no approved treatments directly address the underlying genetic causes of CPVT.

目前，尚无获批的治疗方法可以直接解决CPVT的潜在遗传原因。

SGT-501 is an adeno-associated virus (AAV)-based gene therapy designed to deliver a functional, full-length, codon-optimised version of the human cardiac calsequestrin (CASQ2) gene directly to heart muscle cells. This approach is aimed at correcting the calcium handling problems and abnormal heart rhythms seen in individuals with CPVT, particularly those caused by RYR2 gene instability..

SGT-501是一种基于腺相关病毒（AAV）的基因疗法，旨在将功能性、全长、密码子优化的人类心脏钙网蛋白（CASQ2）基因直接递送到心肌细胞。这种方法旨在纠正CPVT患者中的钙处理问题和异常心律，特别是那些由RYR2基因不稳定性引起的症状。

SGT-501 also received Investigational New Drug (IND) clearance from the FDA and Clinical Trial Application (CTA) approval from Health Canada on 8 July 2025. A Phase 1b trial is expected to begin in the final quarter of 2025. This first-in-human study will assess the safety, tolerability, and potential effectiveness of the gene therapy in individuals living with CPVT..

SGT-501还于2025年7月8日获得了美国食品药品监督管理局（FDA）的新药临床试验（IND）批准和加拿大卫生部的临床试验申请（CTA）批准。预计1b期试验将在2025年最后一个季度开始。这项首次人体研究将评估基因疗法在CPVT患者中的安全性、耐受性和潜在有效性。

The therapy is being developed to treat catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare and potentially life-threatening inherited heart condition.

该疗法正在开发用于治疗儿茶酚胺能多形性室性心动过速 (CPVT)，这是一种罕见且可能危及生命的遗传性心脏病。

The Fast Track designation is intended to support the development of treatments for serious conditions with unmet medical needs.

快速通道指定旨在支持针对未满足医疗需求的严重病症的治疗方法的开发。

It allows for more frequent communication with the FDA and may lead to a faster review process.

它允许与 FDA 更频繁地沟通，并可能加快审查进程。