EVRY, France--(BUSINESS WIRE)--In recognition of LGMD Awareness Day today, Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies, announced the enrollment of the last patient in the dose-escalation phase of its Phase 1b clinical trial of ATA-100, a gene therapy for patients with limb-girdle muscular dystrophy Type 2I/R9 (LGMD-2I/R9, protocol code ATA-001-FKRP)..

法国埃夫里——（商业新闻短讯）——为了表彰今天的LGMD意识日，专注于开发针对肌营养不良症和心肌病的新一代基因疗法的临床阶段生物技术公司Atamyo Therapeutics宣布将最后一名患者纳入其针对肢带型肌营养不良症2I/R9型（LGMD-2I/R9，协议代码ATA-001-FKRP）患者的基因疗法ATA-100 1b期临床试验的剂量递增阶段。。

“The first patients dosed with ATA-100 have experienced promising functional results and the gene therapy has been overall well tolerated in all treated patients with no unexpected safety signal,” said Atamyo CEO and Co-Founder Stephane Degove. “Updated results of the clinical trial will be featured in an oral presentation at the 29th International Annual Congress of the World Muscle Society (WMS) in Prague, October 8 - 12, 2024.”.

Atamyo首席执行官兼联合创始人斯蒂芬·德戈夫（StephaneDegove）表示：“首批服用ATA-100的患者取得了令人鼓舞的功能结果，基因治疗在所有接受治疗的患者中总体耐受性良好，没有意外的安全信号。”。“该临床试验的最新结果将于2024年10月8日至12日在布拉格举行的第29届世界肌肉学会（WMS）国际年会上发表。”。

Atamyo also announced the filing of an Investigational New Drug (IND) application with the US FDA for a Phase 1b/2b clinical trial of its gene therapy, ATA-200, for patients with LGMD-2C/R5. The deployment in the US of Atamyo’s clinical trial of ATA-200 (NCT05973630) is funded by The Dion Foundation for Children with Rare Diseases..

Atamyo还宣布向美国FDA提交一份研究性新药（IND）申请，用于LGMD-2C/R5患者的基因治疗ATA-200的1b/2b期临床试验。Atamyo的ATA-200临床试验（NCT05973630）在美国的部署由迪翁罕见病儿童基金会资助。。

The multicenter Phase1b open-label dose escalation study will evaluate safety, pharmacodynamics, efficacy, and immunogenicity in children receiving intravenous ATA-200, a single-dose Adeno-Associated Virus (AAV) vector carrying the human γ-sarcoglycan transgene. This study has already received regulatory clearance in France and Italy..

多中心1B期开放标签剂量递增研究将评估接受静脉注射ATA-200（一种携带人γ-糖聚糖转基因的单剂量腺相关病毒（AAV）载体）的儿童的安全性，药效学，疗效和免疫原性。这项研究已经在法国和意大利获得监管许可。。

In addition, Atamyo announced the US FDA has awarded Rare Pediatric Disease Designation for ATA-200. The designation was requested based on the potential for ATA-200 to address an unmet medical need for LGMD-2C/R5, a serious and debilitating condition that affects children and leads to loss of ambulation before adulthood..

此外，Atamyo宣布美国FDA已授予ATA-200罕见儿科疾病称号。该名称是根据ATA-200的潜力提出的，以解决LGMD-2C/R5的医疗需求未得到满足，LGMD-2C/R5是一种严重且衰弱的疾病，会影响儿童并导致成年前无法行走。。

“We are thrilled to have completed the recruitment of the dose escalation phase of our Phase 1b study of ATA-100. Data of the dose escalation will serve at selecting the dose to carry over in the pivotal phase of the study,” said Sophie Olivier, MD, Chief Medical Officer of Atamyo. “We look forward to presenting the preliminary results of the first dose cohort at the forthcoming WMS Congress and at American Society of Gene & Cell Therapy’s Breakthroughs in Muscular Dystrophy Conference Nov.

“我们很高兴完成了ATA-100 1b期研究剂量递增阶段的招募。剂量递增的数据将有助于选择在研究的关键阶段进行的剂量，”Atamyo首席医疗官SophieOlivier医学博士说。“我们期待着在即将举行的WMS大会和11月举行的美国基因与细胞治疗学会肌肉萎缩症突破会议上介绍第一个剂量队列的初步结果。

19 – 20, 2024 in Chicago.”.

2024年19-20日，芝加哥。”。

LGMD-2I/R9 is a rare genetic disease caused by mutations in the gene that produces fukutin-related protein (FKRP). It affects an estimated 5,000 people in the US and Europe. In the most common form, symptoms appear around late childhood or early adulthood. Patients suffer from progressive muscular weakness leading to loss of ambulation.

LGMD-2I/R9是一种罕见的遗传疾病，由产生福库汀相关蛋白（FKRP）的基因突变引起。它影响了美国和欧洲大约5000人。最常见的症状出现在儿童晚期或成年早期。患者患有进行性肌无力，导致无法行走。

They are also prone to respiratory impairment. There is currently no curative treatment for LGMD2I/R9..

他们也容易出现呼吸障碍。目前尚无LGMD2I/R9的治疗方法。。

ATA-100, a single-administration gene therapy candidate for LGMD-2I/R9, delivers a normal copy of the gene for production of FKRP proteins. The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at CNRS who heads the Progressive Muscular Dystrophies Laboratory at Genethon..

ATA-100是LGMD-2I/R9的单次给药基因治疗候选物，可提供该基因的正常拷贝以产生FKRP蛋白。该疗法基于Atamyo首席科学官Isabelle Richard博士的研究，Isabelle Richard博士是CNRS的研究主任，他是Genethon进行性肌营养不良实验室的负责人。。

LGMD-2C/R5 is a rare genetic disease caused by mutations in the gene that produces γ-sarcoglycan, a transmembrane protein that is involved in the connection between muscle fibers and their environment. It affects an estimated 2,000 people in Europe. In the classical form, symptoms appear in early childhood and patients suffer from progressive muscular weakness leading to loss of ambulation before adulthood.

LGMD-2C/R5是一种罕见的遗传疾病，由产生γ-糖聚糖的基因突变引起，γ-糖聚糖是一种跨膜蛋白，参与肌肉纤维与其环境之间的连接。它影响了欧洲大约2000人。在经典形式中，症状出现在儿童早期，患者患有进行性肌肉无力，导致成年前无法行走。

Cardiac involvement, typically presenting as dilated cardiomyopathy is reported in approximately half of patients. There is no curative treatment. The management of LGMD-2C/R5 is solely supportive..

据报道，大约一半的患者心脏受累，通常表现为扩张型心肌病。没有治愈的方法。LGMD-2C/R5的管理完全是支持性的。。

ATA-200, the gene therapy for LGMD-2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan. In preclinical mice models, ATA-200 demonstrated its tolerability and capability to correct symptoms and biomarkers of the pathology. Atamyo plans to initiate dosing in patients for ATA-200 in the 4th quarter of 2024..

ATA-200是LGMD-2C/R5的基因疗法，可提供该基因的正常拷贝以产生γ-糖聚糖。在临床前小鼠模型中，ATA-200证明了其耐受性和纠正病理症状和生物标志物的能力。Atamyo计划在2024年第四季度开始给患者服用ATA-200。。

In addition to its LGMD-2C/R5 and LGMD-2I/R9 gene therapies, Atamyo is in IND-enabling studies for LGMD-2A/R1, which is related to deficiencies in calpain3 protein.

除了其LGMD-2C/R5和LGMD-2I/R9基因疗法外，Atamyo还在进行针对LGMD-2A/R1的IND研究，这与钙蛋白酶3蛋白的缺陷有关。

About Atamyo Therapeutics

关于Atamyo Therapeutics

Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon.

Atamyo Therapeutics是一种临床阶段的生物制药，专注于开发新一代有效且安全的神经肌肉疾病基因疗法。Atamyo是基因治疗先驱Genethon的衍生产品，利用Genethon进行性肌营养不良实验室在基于AAV的基因治疗和肌营养不良方面的独特专业知识。

Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with two clinical-stage programs targeting respectively LGMD-R9 and LGMD-R5. The name of the company is derived from two words: Celtic Atao which means “Always” or “Forever” and Myo which is the Greek root for muscle.

Atamyo最先进的计划针对不同形式的肢带型肌营养不良症（LGMD），其中两个临床阶段计划分别针对LGMD-R9和LGMD-R5。该公司的名称来源于两个词：凯尔特人的Atao，意思是“永远”或“永远”，Myo是希腊语muscle的词根。

Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit www.atamyo.com.

Atamyo传达了其承诺的精神，即通过终身有效的治疗来改善受神经肌肉疾病影响的患者的生活。有关更多信息，请访问www.atamyo.com。