Rocket Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for RP-A701, a gene therapy candidate for the treatment of BAG3-associated Dilated Cardiomyopathy (BAG3-DCM).

Rocket Pharmaceuticals 宣布，美国食品药品监督管理局 (FDA) 已批准其用于治疗 BAG3 相关扩张型心肌病 (BAG3-DCM) 的基因疗法候选药物 RP-A701 的新药临床试验 (IND) 申请。

BAG3-associated Dilated Cardiomyopathy is a rare genetic condition caused by mutations in the BAG3 gene. These mutations disrupt the production and function of the BAG3 protein, which plays a critical role in cardiac contractility, structural integrity, protein regulation, and cell survival processes.

BAG3相关的扩张型心肌病是一种由BAG3基因突变引起的罕见遗传疾病。这些突变破坏了BAG3蛋白的生成及其功能，该蛋白在心脏收缩性、结构完整性、蛋白质调节和细胞存活过程中起着关键作用。

The resulting protein malfunction leads to the accumulation of damaged proteins in heart cells, which impairs cardiac function and can lead to early-onset heart failure..

由此产生的蛋白质功能障碍会导致心肌细胞中受损蛋白质的积累，这会损害心脏功能并可能导致早发性心力衰竭。

Initial participants will include individuals with implantable cardioverter defibrillators (ICDs) who are at high risk of worsening heart failure and cardiac-related death. The trial will monitor BAG3 protein expression, changes in cardiac biomarkers, and key indicators of disease progression.

初始参与者将包括植入式心律转复除颤器（ICD）的高危心力衰竭恶化和心脏相关死亡风险的个体。试验将监测BAG3蛋白表达、心脏生物标志物的变化以及疾病进展的关键指标。

The therapy is based on the AAVrh.74 viral vector and targets a severe inherited form of heart failure.

该疗法基于 AAVrh.74 病毒载体，针对一种严重的遗传性心力衰竭。

RP-A701 becomes the company’s third clinical-stage gene therapy within its cardiovascular portfolio. The upcoming first-in-human Phase 1 clinical trial will focus on adults with advanced BAG3-DCM. The trial, set to begin soon, will be conducted across multiple sites and will use a dose-escalation approach.

RP-A701成为该公司心血管产品组合中的第三个临床阶段基因疗法。即将进行的首次人体一期临床试验将专注于患有晚期BAG3-DCM的成人患者。该试验预计很快开始，将在多个地点进行，并采用剂量递增的方法。

The primary aim is to assess the safety, biological activity, and early signs of efficacy following a single administration of RP-A701..

主要目的是评估单次给予RP-A701后的安全性、生物活性和早期疗效迹象。

It is estimated that up to 30,000 individuals in the United States may be affected by this condition. Current treatments, including ICDs, resynchronisation devices, and heart transplants, may help manage symptoms but often fail to halt disease progression or address the root genetic cause.

据估计，美国可能有多达30,000人受此疾病影响。目前的治疗方法，包括植入式心脏复律除颤器（ICD）、再同步装置和心脏移植，可能有助于缓解症状，但往往无法阻止疾病进展或解决根本的遗传原因。

Rocket’s investigational therapy aims to target the underlying mutation and provide a long-term therapeutic solution.

火箭公司的研究性疗法旨在针对根本的基因突变，并提供长期的治疗解决方案。