PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 Phase 1/2 clinical trial in Spinal Muscular Atrophy (SMA) Type I at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore, Maryland on May 8, 2024 (poster #627)..

费城--（商业新闻短讯）--Exegenesis Bio是一家快速发展的全球基因药物公司，很高兴宣布其EXG001-307 I型脊髓性肌萎缩症（SMA）1/2期临床试验的临床疗效和安全性数据将于2024年5月8日在马里兰州巴尔的摩举行的美国基因与细胞治疗学会（ASGCT）年会上发布（海报#627）。。

EXG001-307 is a next generation recombinant adeno-associated virus (rAAV) gene therapy in development for Spinal Muscular Atrophy (SMA) Type 1. Data from nine patients demonstrate improved head control and sitting without external assistance as early as three months following dosing.

EXG001-307是正在开发用于1型脊髓性肌萎缩症（SMA）的下一代重组腺相关病毒（rAAV）基因疗法。来自9名患者的数据表明，早在给药后三个月，头部控制和坐姿就得到了改善，没有外部帮助。

EXG001-307 has a unique AAV design, including a novel pro-NS promoter, leading to high expression in target spinal cord tissue and reduced off target expression in liver and heart tissue versus currently available gene therapy.

EXG001-307具有独特的AAV设计，包括一种新型的pro-NS启动子，与目前可用的基因治疗相比，可在靶脊髓组织中高表达，并降低肝脏和心脏组织中的脱靶表达。

Data Highlights:

数据亮点：

EXG001-307 has a unique engineered AAV design that includes a novel pro-NS promoter, which is believed to contribute to improved efficacy and safety in SMA Type 1.

EXG001-307具有独特的工程AAV设计，其中包括一种新型pro-NS启动子，据信有助于提高SMA 1型的功效和安全性。

EXG001-307 has demonstrated high expression in target spinal cord tissue and reduced off target expression in liver and heart tissue than AAV9.CBA.SMN1.

与AAV9.CBA相比，EXG001-307在靶脊髓组织中高表达，在肝脏和心脏组织中脱靶表达降低。SMN1。

Patients in the EXG001-307 low-dose group (1.1 E 14 vg/kg) achieved head control 3 to 6 months after dosing and sitting without external assistance 11 months following dosing.

EXG001-307低剂量组（1.1 E 14 vg/kg）的患者在给药后3至6个月达到头部控制，并在给药后11个月在没有外部帮助的情况下坐着。

The first patient in the mid-dose group (1.5 E 14 vg/kg) achieved sitting with assistance 3 months following dosing.

中剂量组的第一名患者（1.5 E 14 vg/kg）在给药后3个月在协助下坐着。

EXG001-307 demonstrated high tolerability and no dose-limiting toxicity, no >Grade 2 test article related serious adverse events, and no >Grade 1 elevation of transaminases or cardiac enzymes.

EXG001-307表现出高耐受性，无剂量限制性毒性，无>2级供试品相关的严重不良事件，转氨酶或心肌酶无>1级升高。

“Spinal Muscular Atrophy is a debilitating and fatal genetic disease that impacts the lives of thousands of babies and young children and their families. I am excited that our team is making rapid progress in our mission to bring this much-needed treatment option to SMA patients worldwide. We are excited about the clinical efficacy and safety data emerging from our Phase 1/2 SMA clinical trial in China.

“脊髓性肌萎缩症是一种使人衰弱且致命的遗传疾病，影响着数千名婴幼儿及其家人的生活。我很高兴我们的团队在为全球SMA患者提供这种急需的治疗选择的任务中取得了快速进展。我们对中国1/2期SMA临床试验的临床疗效和安全性数据感到兴奋。

This is leading us to accelerate development in the US. We expect to file our US IND for SMA Type 1 in 4Q 2024. We are also exploring an accelerated development path in the slightly older SMA Type 2/3 patient population. We look forward to sharing further details during our poster session at ASGCT in Baltimore on May 8, 2024.

这使我们加快了在美国的发展。我们预计将于2024年第4季度提交SMA 1型的美国IND。我们还正在探索年龄稍大的2/3型SMA患者群体的加速发展道路。我们期待着在2024年5月8日于巴尔的摩举行的ASGCT海报会议上分享更多细节。

I would like to congratulate our team for embracing our mission to bring innovative and life-changing genetic medicines to patients globally and for driving our ambitious clinical development timelines,” stated Dr. Zhenhua Wu, CEO of Exegenesis Bio..

Exegenesis Bio首席执行官吴振华博士表示：“我要祝贺我们的团队接受我们的使命，为全球患者带来创新和改变生命的遗传药物，并推动我们雄心勃勃的临床开发时间表。”。。

About EXG001-307

关于EXG001-307

EXG001-307 is a recombinant adeno-associated virus (rAAV)-based gene therapy in clinical development for Spinal Muscular Atrophy (SMA) Type 1. EXG001-307 has demonstrated significantly improved efficacy in SMA Type 1 patients (CHOP Intend score) and lower off target effects in liver and cardiac tissue.

EXG001-307是一种基于重组腺相关病毒（rAAV）的基因疗法，用于1型脊髓性肌萎缩症（SMA）的临床开发。EXG001-307在SMA 1型患者中显示出显着改善的疗效（CHOP意向评分），并降低了肝脏和心脏组织的脱靶效应。

Patients in both low and mid-dose cohorts demonstrated improved head control and sitting ability within the 3 to 11 months of dosing. Exegenesis Bio will share additional clinical and non-clinical efficacy and safety data during a poster session at ASGCT on Wednesday May 8, 2024. Poster # 627..

低剂量组和中剂量组的患者在给药的3至11个月内表现出改善的头部控制和坐姿能力。Exegenesis Bio将于2024年5月8日（星期三）在ASGCT的海报会议上分享更多的临床和非临床疗效和安全性数据。海报#627。。

About Spinal Muscular Atrophy

关于脊髓性肌萎缩症

Spinal Muscular Atrophy (SMA) is an autosomal recessive inherited neuromuscular disease, characterized by motor neuron degeneration, skeletal muscle atrophy, muscle weakness, and ultimately death. SMA is the most common fatal disease in infants, with incidence in newborns ranging from one in six thousand to one in ten thousand..

脊髓性肌萎缩症（SMA）是一种常染色体隐性遗传性神经肌肉疾病，其特征是运动神经元变性，骨骼肌萎缩，肌肉无力，最终死亡。SMA是婴儿中最常见的致命疾病，新生儿的发病率从六千分之一到一万分之一不等。。

SMA is classified into five subtypes, based on the age of onset, severity of symptoms, and motor milestones. SMA Type 0 manifests prenatally, Type 1 appears during the first six months of age, Type 2 appears between 6 and 18 months of age, Type 3 begins as early as 18 months of age, while Type 4 has late onset and symptoms develop in adulthood1.

根据发病年龄，症状严重程度和运动里程碑，SMA分为五种亚型。SMA 0型在产前表现，1型出现在前六个月，2型出现在6至18个月之间，3型早在18个月大时开始，而4型发病较晚，症状在成年后发展1。

Data suggest that infants with SMA type 1, the most common type, have a median survival of 10.5 months, and 8% survival rate to 20 months without permanent respiratory support2..

数据表明，最常见的SMA 1型婴儿的中位生存期为10.5个月，无永久性呼吸支持的20个月生存率为8%。。

SMA is caused by a homozygous loss of SMN1 genes on chromosome 5q13 due to mutations or deletions, leading to a decreased level of survival motor neuron (SMN) protein, which triggers motor neuron overexcitation and degeneration, synaptic dysfunction, and reduced muscle fiber volume. Current SMA therapies work by increasing levels of SMN protein in the body3..

SMA是由突变或缺失导致的5q13号染色体上SMN1基因的纯合丢失引起的，导致存活运动神经元（SMN）蛋白水平降低，从而引发运动神经元过度兴奋和变性，突触功能障碍和肌纤维体积减少。目前的SMA疗法通过增加体内SMN蛋白的水平来起作用3。。

About Exegenesis Bio

关于Exenesis Bio

Exegenesis Bio is a clinical stage global gene therapy company with operations in USA and China. The company’s innovative gene therapy pipeline is based on proprietary capsids, promoters and unique protein engineering designs. The company has received IND clearances from US FDA and China CDE and is currently conducting Phase 1/2 clinical trials in USA and China..

Exegenesis Bio是一家临床阶段的全球基因治疗公司，在美国和中国运营。该公司的创新基因治疗管道基于专有衣壳，启动子和独特的蛋白质工程设计。该公司已获得美国FDA和中国CDE的IND许可，目前正在美国和中国进行1/2期临床试验。。

EXG001-307 is a recombinant AAV (rAAV) gene therapy in Phase 1/2 clinical development for Spinal Muscular Atrophy Type 1 in China. The company plans to file a US IND for EXG001-307 in 2024.

EXG001-307是中国1型脊髓性肌萎缩症1/2期临床开发中的重组AAV（rAAV）基因疗法。该公司计划在2024年为EXG001-307提交美国IND。

EXG102-031 is a recombinant AAV (rAAV) based gene therapy in clinical development for neovascular Age Related Macular Degeneration (nAMD) in USA and China. The EXG102-031 clinical study is being conducted at two clinical sites in the USA and eight additional sites in China.

EXG102-031是一种基于重组AAV（rAAV）的基因疗法，用于美国和中国新生血管性年龄相关性黄斑变性（nAMD）的临床开发。EXG102-031临床研究正在美国的两个临床地点和中国的另外八个地点进行。

Exegenesis Bio has built full end-to-end capabilities that include discovery, translational, non-clinical and clinical development, quality and manufacturing. The company operates state-of-the-art cGMP manufacturing facilities that include 500 Liter and 2,000 Liter disposable bioreactors for viral vectors and 30 Liter disposable fermenters for plasmids.

Exegenesis Bio建立了完整的端到端能力，包括发现，转化，非临床和临床开发，质量和制造。该公司拥有最先进的cGMP制造设施，包括用于病毒载体的500升和2000升一次性生物反应器以及用于质粒的30升一次性发酵罐。

The company has raised over $200 Million since inception in 2019 and currently employs over 200 scientific and operations staff worldwide..

自2019年成立以来，该公司已筹集了2亿多美元，目前在全球范围内雇用了200多名科学和运营人员。