Klotho Neurosciences, Inc. today announced that it is moving forward with manufacturing and process development work in preparation for clinical trials of KLTO-202, its investigational gene therapy for amyotrophic lateral sclerosis (ALS).

克洛托神经科学公司今天宣布，它正在推进 KLTO-202 的制造和工艺开发工作，为这款用于肌萎缩侧索硬化症（ALS）的在研基因疗法的临床试验做准备。

A unique RNA splice variant of the human gene called alpha-Klotho has been licensed by the Company from the Autonomous University of Barcelona ('UAB') including patents, patent applications, research, knowhow and other intellectual properties for use in the development of advanced gene and gene-engineered cell therapies.

公司已从巴塞罗那自治大学（“UAB”）获得了一种名为α-Klotho的人类基因的独特RNA剪接变体的许可，包括专利、专利申请、研究、技术诀窍及其他知识产权，用于开发先进的基因和基因工程细胞疗法。

The human alpha-Klotho gene is located in cells found throughout the human body. It is a five exon gene that produces two protein isoforms: a full-length protein found mainly in cells of the kidney called membrane-bound Klotho (or m-KL), which controls phosphate homeostasis, and a much smaller protein isoform called secreted alpha-Klotho (or s-KL).

人类的α-Klotho基因位于人体各处的细胞中。它是一个包含五个外显子的基因，产生两种蛋白质异构体：一种是主要在肾脏细胞中发现的全长蛋白，称为膜结合型Klotho（或m-KL），其控制磷的稳态；另一种是较小的蛋白质异构体，称为分泌型α-Klotho（或s-KL）。

The s-KL RNA splice variant is found mainly in the brain and spinal cord neurons; the variant protein is neuroprotective by minimizing both oxidative stress and neuroinflammation. Animal studies supported by the Company over the past two years in mouse and non-human primate models of rapid aging, in models of human Alzheimer's disease, and in models of ALS have shown that over-expression and amplification of the tissue levels of s-KL using a gene therapy approach result in highly favorable therapeutic outcomes in every model tested.

s-KL RNA剪接变体主要存在于脑和脊髓神经元中；该变异蛋白通过减少氧化应激和神经炎症发挥神经保护作用。公司在过去两年中支持的动物研究，在快速衰老的小鼠和非人灵长类模型、人类阿尔茨海默病模型以及ALS模型中表明，利用基因疗法过表达并提高组织中的s-KL水平，在所有测试模型中均产生了非常有利的治疗效果。

These results have been published in peer-reviewed scientific journals and now support the transition of KLTO-202 into the clinical development stage..

这些结果已发布在经同行评审的科学期刊上，现在支持 KLTO-202 过渡到临床开发阶段。

The Company expects that it will take approximately eight months to complete process development and manufacturing of KLTO-202, and about four to six months to conduct meetings with FDA, complete all FDA-mandated animal safety studies, file an investigational new drug application (IND), train and prepare clinical sites where the Phase I/II studies can be conducted, and then begin the single-dose gene therapy studies in ALS patients by the third quarter of next year.

公司预计，完成KLTO-202的工艺开发和生产大约需要八个月，并且大约需要四到六个月的时间与FDA召开会议、完成所有FDA要求的动物安全性研究、提交新药临床试验申请（IND）、培训并准备可进行I/II期研究的临床试验点，然后在明年第三季度开始对ALS患者进行单剂量基因治疗研究。

The Company will work with contract research organizations (CROs) to facilitate all activities including manufacturing and clinical trials without the need to hire several dozen staff members, which would significantly increase our operating overhead..

公司将会与合同研究组织（CRO）合作，以促进包括制造和临床试验在内的所有活动，而无需雇佣几十名员工，这将大大增加我们的运营成本。

Dr. Joseph Sinkule, the Company's CEO and founder commented: 'With our recent fundraising success, we're moving forward with manufacturing the s-KL transgene DNA for KLTO-202. We've identified a more efficient method of producing the AAV vector to deliver the s-KL gene directly to motor neurons—the cells most affected by ALS.

公司首席执行官兼创始人约瑟夫·辛库勒博士评论道：“随着我们最近的融资成功，我们正在推进生产KLTO-202的s-KL转基因DNA。我们已经找到了一种更高效的方法来生产AAV载体，以将s-KL基因直接递送到运动神经元——这是受ALS影响最严重的细胞。”

Our goal is to increase local s-KL protein levels to protect these neurons from the damage that leads to voluntary and involuntary muscle paralysis and ultimately death.'.

我们的目标是增加局部 s-KL 蛋白水平，以保护这些神经元免受导致随意和不随意肌肉瘫痪并最终死亡的损伤。

ALS typically progresses rapidly, with most patients losing mobility, respiratory function, and life within just 2–3 years of diagnosis.

肌萎缩侧索硬化症通常进展迅速，大多数患者在确诊后 2-3 年内失去行动能力、呼吸功能和生命。

About Klotho Neurosciences, Inc.

关于克洛托神经科学公司

Klotho Neurosciences, Inc. (NASDAQ: KLTO), is a biogenetics company focused on the development of innovative, disease-modifying cell and gene therapies using a protein derived from a patented form of the 'anti-aging' human Klotho gene (s-KL), and its novel delivery systems to transform and improve the treatment of neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease.

克洛托神经科学公司（纳斯达克股票代码：KLTO）是一家生物遗传学公司，专注于利用源自专利形式的“抗衰老”人类克洛托基因（s-KL）的蛋白质，开发创新的疾病修饰细胞和基因疗法，并通过其新型递送系统彻底改变和改善对肌萎缩侧索硬化症、阿尔茨海默病和帕金森病等神经退行性疾病和年龄相关疾病的治疗。

The Company's current portfolio consists of its proprietary cell and gene therapy programs using DNA and RNA as therapeutics and genomics-based diagnostic assays. The Company is managed by a team of individuals and advisors who are highly experienced in biopharmaceutical product development and commercialization..

公司目前的产品组合包括其使用DNA和RNA作为治疗剂的专有细胞和基因治疗计划，以及基于基因组学的诊断分析。公司由在生物制药产品开发和商业化方面经验丰富的个人和顾问团队管理。

Investor Contact and Corporate Communications - Jeffrey LeBlanc, CFO

投资者联系和企业传播 - 杰弗里·勒布朗，首席财务官

ir@klothoneuro.com

ir@klothoneuro.com

Website: www.klothoneuro.com

网站：www.klothoneuro.com

Forward-Looking Statements:

前瞻性声明：

This press release contains forward-looking statements. These statements are made under the 'safe harbor' provisions of the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements generally are identified by the words 'believe,' 'project,' 'expect,' 'anticipate,' 'estimate,' 'intend,' 'strategy,' 'future,' 'opportunity,' 'plan,' 'may,' 'should,' 'will,' 'would,' 'will be,' 'will continue,' 'will likely result,' and similar expressions.

本新闻稿包含前瞻性陈述。这些陈述是根据美国1995年私人证券诉讼改革法案的“安全港”条款作出的。这些前瞻性陈述通常可以通过诸如“相信”、“预计”、“预测”、“预期”、“估计”、“意图”、“策略”、“未来”、“机会”、“计划”、“可能”、“应该”、“将”、“将会”、“将继续”、“预计将导致”以及类似表述来识别。

Without limiting the generality of the foregoing, the forward-looking statements in this press release include descriptions of the Company's future commercial operations. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties.

在不限制前述内容的一般性前提下，本新闻稿中的前瞻性陈述包括对公司未来商业运营的描述。前瞻性陈述是基于当前预期和假设对未来事件的预测、推断及其他陈述，因此存在风险和不确定性。

Many factors could cause actual future events to differ materially from the forward-looking statements in this press release, such as the Company's inability to implement its business plans, identify and realize additional opportunities, or meet or exceed its financial projections and changes in the regulatory or competitive environment in which the Company operates.

许多因素可能导致实际未来事件与此新闻稿中的前瞻性声明存在重大差异，例如公司无法实施其业务计划、识别并实现额外的机会，或达到或超出其财务预测，以及公司运营所处的监管或竞争环境的变化。

You should carefully consider the foregoing factors and the other risks and uncertainties described in the documents filed or to be filed by the Company with the U.S. Securities and Exchange Commission (the 'SEC') from time to time, which could cause actual events and results to differ materially from those contained in the forward-looking statements.

您应当仔细考虑上述因素以及公司不时向美国证券交易委员会（“SEC”）提交或将会提交的文件中描述的其他风险和不确定性，这些因素可能导致实际事件和结果与前瞻性陈述中包含的内容有重大差异。

All information provided herein is as of the date of this press release, and the Company undertakes no obligation to update any forward-looking statement, except as required under applicable law..

本新闻稿中的所有信息均为截至本新闻稿发布之日的信息，公司不承担更新任何前瞻性声明的义务，除非适用法律要求。

Source: prnewswire.com

来源：prnewswire.com