- VQ-101 is a potent allosteric activator of glucocerebrosidase (GCase)

-VQ-101是葡萄糖脑苷脂酶（GCase）的有效变构激活剂

- The Phase 1 program aims to demonstrate VQ-101’s pharmacodynamic effects in healthy volunteers and Parkinson’s patients

-第一阶段计划旨在证明VQ-101在健康志愿者和帕金森病患者中的药效学作用

CHICAGO, April 09, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio, a clinical-stage biopharmaceutical company dedicated to discovering and developing next-generation medicines for the treatment of neurodegenerative diseases, announced that the first patient has been dosed in a first-in-human Phase 1 clinical study evaluating VQ-101 in healthy individuals and patients with various forms of Parkinson’s disease (PD).

芝加哥，2024年4月9日（环球通讯社）--Vanqua Bio是一家临床阶段的生物制药公司，致力于发现和开发用于治疗神经退行性疾病的下一代药物，该公司宣布，第一名患者已在一项针对健康个体和各种形式帕金森氏病（PD）患者评估VQ-101的首次人体1期临床研究中服用。

VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase)..

VQ-101是溶酶体酶葡萄糖脑苷脂酶（GCase）的口服脑渗透性小分子变构激活剂。。

“VQ-101 demonstrated promising efficacy, safety, pharmacokinetics, and target engagement in preclinical studies. The launch of the Phase 1 study exemplifies Vanqua Bio's ability to translate our lysosomal biology and medicinal chemistry expertise into pioneering treatments that are well suited for clinical development,” said Jim Sullivan, PhD, Chief Executive Officer of Vanqua Bio.

Vanqua Bio首席执行官Jim Sullivan博士说：“VQ-101在临床前研究中显示出良好的疗效、安全性、药代动力学和靶向参与。第一阶段研究的启动证明了Vanqua Bio能够将我们的溶酶体生物学和药物化学专业知识转化为非常适合临床开发的开创性治疗方法。”。

'This evolution to a clinical-stage company is an important milestone for Vanqua and brings us closer to our goal of addressing unmet patient needs through evidence-based innovation.”.

“这种向临床阶段公司的演变是Vanqua的一个重要里程碑，使我们更接近通过循证创新解决未满足患者需求的目标。”。

Vanqua is initially developing VQ-101 for the treatment of Parkinson’s disease with mutations in GBA1 (GBA-PD), the gene that encodes GCase. Mutations in GBA1 are the most common genetic risk factor for PD, representing approximately 10% of patients with PD worldwide. Mutations in the GBA1 gene result in a decrease in the activity of GCase.

Vanqua最初正在开发VQ-101，用于治疗GBA1（GBA-PD）突变的帕金森病，GBA1是编码GCase的基因。GBA1突变是PD最常见的遗传危险因素，约占全球PD患者的10%。GBA1基因突变导致GCase活性降低。

Reductions in the activity of GCase disrupt the function of lysosomes, the recycling centers of the cells, enabling toxic forms of proteins, including alpha synuclein, to accumulate and harm neurons..

GCase活性的降低破坏了溶酶体的功能，溶酶体是细胞的再循环中心，使有毒形式的蛋白质（包括α-突触核蛋白）积累并伤害神经元。。

In developing VQ-101, Vanqua is adopting a precision-medicine approach that focuses initially on patients with GBA-PD, the largest genetically defined segment of PD. In preclinical studies, Vanqua has demonstrated that VQ-101 activates lysosomal GCase in a live-cell assay in vitro, ex vivo, and in vivo, demonstrates robust pathway engagement, and blocks the accumulation of insoluble alpha synuclein, which is the pathological hallmark of PD..

在开发VQ-101的过程中，Vanqua采用了一种精准医学方法，该方法最初专注于PD最大的基因定义片段GBA-PD患者。在临床前研究中，Vanqua已经证明VQ-101在体外，离体和体内活细胞测定中激活溶酶体GCase，表现出强大的途径参与，并阻止不溶性α-突触核蛋白的积累，这是PD的病理标志。。

About the Phase 1 Study

关于第一阶段研究

The first-in-human study with VQ-101 will be a randomized, double-blind, placebo-controlled single- and multiple-ascending dose study in healthy volunteers and PD patients with or without GBA1 mutations. The study is designed to evaluate VQ-101’s safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD)..

VQ-101的首次人体研究将是一项随机，双盲，安慰剂对照的单剂量和多剂量递增研究，对象是健康志愿者和有或没有GBA1突变的PD患者。该研究旨在评估VQ-101的安全性和耐受性，药代动力学（PK）和药效学（PD）。。

About Vanqua Bio

关于Vanqua Bio

Founded in 2019 and headquartered in Chicago, Vanqua Bio is a biopharmaceutical company dedicated to discovering and developing next-generation medicines that have the potential to transform the lives of patients with neurodegenerative diseases. Our technology platform utilizes human genetics and patient-derived neuronal cells to identify, validate, and clinically translate novel disease pathways associated with lysosomal dysfunction or aberrant activation of the innate immune system.

Vanqua Bio成立于2019年，总部位于芝加哥，是一家生物制药公司，致力于发现和开发有可能改变神经退行性疾病患者生活的下一代药物。我们的技术平台利用人类遗传学和患者来源的神经元细胞来识别，验证和临床转化与溶酶体功能障碍或先天免疫系统异常激活相关的新型疾病途径。

Initially, we are targeting glucocerebrosidase (GCase) as a potential treatment for Parkinson’s disease (PD). Additional programs address overactivation of the innate immune system in central and peripheral neurodegenerative disorders, including Alzheimer’s disease. For more information, go to www.vanquabio.com..

最初，我们将葡萄糖脑苷脂酶（GCase）作为帕金森病（PD）的潜在治疗方法。其他计划解决中枢和外周神经退行性疾病（包括阿尔茨海默氏病）中先天免疫系统的过度激活。有关更多信息，请访问www.vanquabio.com。。

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艾丽莎·帕尔多

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