Nuevocor-headquartered biotechnology company developing cures for cardiomyopathies driven by aberrant mechanobiology, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for NVC-001. NVC-001 is an adeno-associated virus (AAV)-based gene therapy designed to treat LMNA-related dilated cardiomyopathy (LMNA DCM).

Nuevocor总部位于-的生物技术公司，专注于开发由异常机械生物学驱动的心肌病治疗方法，今天宣布美国食品和药物管理局（FDA）已批准其新药研究申请（IND），涉及NVC-001。NVC-001是一种基于腺相关病毒（AAV）的基因疗法，旨在治疗与LMNA相关的扩张型心肌病（LMNA DCM）。

Nuevocor plans to initiate an open-label, ascending-dose Phase 1/2 clinical trial of NVC-001 in patients with LMNA DCM..

Nuevocor计划在LMNA DCM患者中启动一项开放标签、剂量递增的1/2期临床试验，以测试NVC-001。

'This IND clearance marks a significant milestone in our mission to develop transformative therapies for patients with genetic cardiomyopathies by leveraging unique insights from our proprietary PrOSIA mechanobiology platform,' said Dr.

“这一IND批准标志着我们在利用专有的PrOSIA机械生物学平台的独特见解，为遗传性心肌病患者开发变革性疗法的使命中，迈出了重要的里程碑。”博士说道。

Yann Chong Tan

颜忠谭

, co-founder and CEO of Nuevocor. 'NVC-001 is the first disease-modifying therapy for LMNA DCM designed to address the underlying mechanobiological root cause of disease. We look forward to initiating our clinical trial to bring this potentially life-changing therapy to patients.'

Nuevocor的联合创始人兼首席执行官表示：“NVC-001是首个针对LMNA DCM的疾病修正疗法，旨在解决疾病的机械生物学根本原因。我们期待启动临床试验，为患者带来这种可能改变生命的疗法。”

LMNA DCM is a serious genetic heart condition caused by mutations in the LMNA gene, which encodes lamin A/C – a protein essential for maintaining nuclear envelope integrity and regulating gene expression in cardiac cells. Mutations in LMNA lead to a weakening of the heart muscle, arrhythmias, and progression to end-stage heart failure.

LMNA DCM 是一种由 LMNA 基因突变引起的严重遗传性心脏病，该基因编码核纤层蛋白 A/C——这种蛋白对维持心肌细胞核膜完整性及调控基因表达至关重要。LMNA 基因突变会导致心肌减弱、心律失常，并发展为终末期心力衰竭。

LMNA DCM is estimated to affect approximately 100,000 patients in the U.S. and .

据估计，LMNA DCM会影响美国大约10万名患者。

Europe

欧洲

, representing a significant unmet medical need.

，代表着一个重要的未满足医疗需求。

NVC-001 is engineered to address the functional mechanical root cause of disease by reducing aberrant mechanical stress on the nucleus, thereby restoring nuclear envelope integrity. Nuclear envelope disruption is a hallmark of LMNA DCM. In preclinical studies, treatment with NVC-001 demonstrated significant benefits, including survival and cardiac function..

NVC-001 旨在通过减少对细胞核的异常机械应力来解决疾病的机械功能根本原因，从而恢复核膜完整性。核膜破坏是 LMNA DCM 的标志。在临床前研究中，使用 NVC-001 治疗显示出显著益处，包括生存率和心脏功能的改善。

The planned Phase 1/2 clinical trial is a first-in-human, 52-week open-label, multicenter, ascending-dose study designed to evaluate the safety, tolerability, and preliminary efficacy of NVC-001 in adult subjects with LMNA-DCM. NVC-001 will be administered as a one-time intravenous infusion to patients in ascending-dose cohorts..

计划进行的 1/2 期临床试验是一项首次人体试验、为期 52 周的开放标签、多中心、剂量递增研究，旨在评估 NVC-001 在 LMNA-DCM 成人患者中的安全性、耐受性和初步疗效。NVC-001 将以一次性静脉输注的方式给予剂量递增队列中的患者。

About Nuevocor

关于Nuevocor

Nuevocor is biotechnology company developing novel therapies for genetic cardiomyopathies driven by aberrant mechanobiology, headquartered in

Nuevocor是一家生物技术公司，致力于开发针对由异常机械生物学驱动的遗传性心肌病的新疗法，总部位于

Singapore

新加坡

with an office in the U.S. and expansion to

在美国设有办事处并扩展到

Europe

欧洲

. Our unique approach, enabled by our proprietary PrOSIA mechanobiology platform, surpasses the limitations of traditional gene replacement therapy – which treats individual gene mutations – to treating defects in shared disease pathways across multiple cardiomyopathies by addressing the functional root cause of disease.

我们独特的方法依托于我们专有的PrOSIA机械生物学平台，超越了传统基因替代疗法的局限性——即治疗单个基因突变——通过解决疾病的机能根本原因，治疗多种心肌病中共有的疾病通路缺陷。

Nuevocor is first-in-disease by addressing genetic cardiomyopathies that are not amendable to gene replacement therapy and have no effective treatment options. (.

Nuevocor 通过针对那些无法通过基因替代疗法治疗且没有有效治疗方案的遗传性心肌病，成为该领域的首创。