SOUTH SAN FRANCISCO, Calif., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that the first patient has been dosed with TN-201 gene therapy for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated HCM in the MyPeak-1 Phase 1b clinical trial at the Cleveland Clinic, Cleveland, Ohio.

南旧金山，加利福尼亚州，2023年10月5日（GLOBE NEWSWIRE）-Tenaya Therapeutics，Inc。（纳斯达克股票代码：TNYA），一家临床阶段生物技术公司，致力于发现，开发和提供潜在的治疗方法解决心脏病的根本原因，今天宣布，在俄亥俄州克利夫兰市克利夫兰诊所的MyPeak-1 1b期临床试验中，第一名患者已接受TN-201基因疗法治疗肌球蛋白结合蛋白C3（MYBPC3）相关的HCM。

Tenaya anticipates sharing initial data from the MyPeak-1 trial in 2024. TN-201 is Tenaya’s potential first-in-class adeno-associated virus (AAV)-based gene therapy designed to deliver a working, full-length copy of the human MYBPC3 gene to heart muscle cells. The working MYBPC3 gene is intended to restore normal levels of myosin-binding protein, which regulates the contraction and relaxation of the heart muscle.

Tenaya预计将于2024年共享MyPeak-1试验的初始数据。TN-201是Tenaya潜在的一流的基于腺相关病毒（AAV）的基因疗法，旨在将人MYBPC3基因的全长工作拷贝提供给心肌细胞。工作的MYBPC3基因旨在恢复正常水平的肌球蛋白结合蛋白，该蛋白调节心肌的收缩和松弛。

In preclinical studies, TN-201 halted disease progression and demonstrated significant and durable disease reversal and survival benefit following a single dose. “MYBPC3 gene mutations are the most common genetic cause of HCM and people with MYBPC3-associated HCM are at increased risk for accelerated decline and serious complications associated with their condition,” said Milind Desai, M.D., MBA, Director at the Cleveland Clinic Hypertrophic Cardiomyopathy Center and Vice Chair, Heart Vascular Thoracic Institute, Cleveland Clinic, and an investigator for the MyPeak-1 Phase 1b clinical trial.

在临床前研究中，TN-201阻止了疾病进展，并且在单次剂量后显示出显着且持久的疾病逆转和存活益处。“MYBPC3基因突变是HCM最常见的遗传原因，与MYBPC3相关的HCM患者加速衰退和与其病情相关的严重并发症的风险增加，”克利夫兰诊所肥厚性梗阻型心肌病中心主任兼克利夫兰诊所心血管胸科研究所副主席Milind Desai博士说，和MyPeak-1 1b期临床试验的研究者。

“TN-201, a gene therapy for MYBPC3-associated HCM, offers the potential of a one-time treatment to correct the underlying genetic cause of disease and improve patient outcomes. We are pleased to participate in the first-in-human clinical trial of TN-201 to explore this new .

“TN-201是一种针对MYBPC3相关HCM的基因治疗方法，它提供了一次性治疗的潜力，可以纠正疾病的潜在遗传原因，改善患者的预后。我们很高兴参加TN-201的首次人体临床试验，以探索这一新发现。