Rocket Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601, its investigational gene therapy for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).

火箭制药公司宣布，美国食品药品监督管理局（FDA）已授予其研究性基因疗法RP-A601用于治疗与斑菲素蛋白-2相关的致心律失常性心肌病（PKP2-ACM）的再生医学先进疗法（RMAT）认定。

RP-A601 is an investigational gene therapy being developed to treat PKP2-related arrhythmogenic cardiomyopathy. It uses a recombinant adeno-associated virus (AAVrh74) to deliver a healthy version of the PKP2 gene. The therapy is administered as a single intravenous infusion and is designed to restore normal levels of plakophilin-2 protein in heart tissue.

RP-A601 是一种正在开发的用于治疗 PKP2 相关心律失常性心肌病的基因疗法。它利用重组腺相关病毒 (AAVrh74) 传递健康的 PKP2 基因。该疗法通过单次静脉输注给药，旨在恢复心脏组织中正常的桥粒斑菲素-2蛋白水平。

RP-A601 holds Fast Track and Orphan Drug designations in both the United States and Europe, recognising its potential as a treatment for a serious, rare condition with limited options..

RP-A601在美国和欧洲均获得了快速通道和孤儿药资格认定，认可了其作为治疗一种严重、罕见且选项有限的疾病的潜力。

PKP2-ACM is a genetic heart disease caused by mutations in the PKP2 gene. It leads to dangerous changes in the heart’s structure and function, including ventricular arrhythmias and a high risk of sudden cardiac death. Around 50,000 people in the US and Europe are estimated to be affected. Current treatments such as medications, implantable cardioverter defibrillators (ICDs), and ablation procedures may help manage symptoms but do not correct the underlying genetic defect.

PKP2-ACM是一种由PKP2基因突变引起的遗传性心脏病。它会导致心脏结构和功能的危险变化，包括室性心律失常和猝死的高风险。据估计，美国和欧洲大约有50,000人受到影响。目前的治疗方法如药物、植入式心律转复除颤器（ICD）和消融手术可能有助于控制症状，但无法纠正潜在的基因缺陷。

These approaches can also be associated with complications, highlighting the need for more effective and long-lasting therapies..

这些方法也可能带来并发症，突显了对更有效和持久疗法的需求。

The designation is based on encouraging early results from a Phase 1 clinical trial.

该指定是基于一期临床试验鼓舞人心的早期结果。

RP-A601 is being studied as a potential one-time treatment using adeno-associated virus (AAV) technology. It aims to address the root genetic cause of PKP2-ACM, a rare and life-threatening inherited heart condition. The disease is marked by abnormal heart rhythms and a high risk of sudden cardiac death..

RP-A601 正在被研究作为一种潜在的一次性治疗方案，使用腺相关病毒 (AAV) 技术。它旨在解决 PKP2-ACM 的根本遗传原因，PKP2-ACM 是一种罕见且危及生命的遗传性心脏病。该疾病的特点是心律异常和高猝死风险。

The RMAT designation is designed to speed up the development of therapies for serious conditions. It provides access to more frequent interactions with the FDA, and offers potential pathways for faster approval based on early clinical data.

RMAT指定旨在加速严重疾病疗法的开发。它提供了与FDA更频繁互动的机会，并基于早期临床数据提供了更快审批的潜在途径。

Preliminary results from the trial presented at a recent scientific meeting showed that three adult patients treated with RP-A601 experienced increased levels of PKP2 protein. Improvements were also observed in structural heart tissue and in clinical measures such as heart function, arrhythmia control, and quality of life.

在最近的一次科学会议上公布的试验初步结果显示，接受RP-A601治疗的三名成年患者的PKP2蛋白水平有所增加。在心脏组织结构和临床指标（如心功能、心律失常控制和生活质量）方面也观察到了改善。

The treatment was generally well tolerated, with no serious safety concerns reported..

治疗通常耐受性良好，未报告严重的安全性问题。

Rocket Pharmaceuticals continues to advance RP-A601 as part of its broader pipeline of gene therapies focused on rare and inherited cardiovascular conditions.

Rocket Pharmaceuticals 继续推进 RP-A601，作为其专注于罕见和遗传性心血管疾病的一系列基因疗法的一部分。