Klotho Neurosciences, Inc., a gene and cell therapy company focused on the treatment of neurodegenerative and other aging-related diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company's novel secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202, or s-KL-AAV.myo) for the treatment of ALS..

Klotho Neurosciences, Inc.，一家专注于治疗神经退行性病变和其他与衰老相关疾病的基因和细胞治疗公司，今天宣布美国食品药品监督管理局（FDA）已授予公司新型分泌型Klotho（s-KL）启动子、基因及递送系统（KLTO-202，或s-KL-AAV.myo）用于治疗ALS的孤儿药资格。

The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. The Orphan Drug Designation provides certain incentives, such as tax credits, toward the cost of human clinical trials and a waiver for the payment of the GDUFA User Fee for market applications.

美国食品药品监督管理局（FDA）授予用于安全有效治疗、诊断或预防在美国影响不到20万人的罕见疾病或病症的药物和生物制品孤儿药资格。孤儿药资格提供某些激励措施，例如针对人体临床试验成本的税收抵免，以及豁免GDUFA用户费用的市场申请支付。

Additionally, Orphan Drug Designation of the product provides the developers seven years of US market exclusivity and independent from the Company's intellectual property protection..

此外，该产品获得孤儿药资格认定后，将为开发者提供七年的美国市场独占期，且独立于公司的知识产权保护。

'Receiving the Orphan Drug Designation for s-KL-AAV.myo for the early treatment of ALS underscores the importance of bringing new treatment options to patients suffering from this rare, universally fatal disease' said Dr. Joseph Sinkule, Klotho's Chief Executive Officer. 'My cousin Karen died from this horrific disease.

“s-KL-AAV.myo获得用于ALS早期治疗的孤儿药资格，突显了为患有这种罕见且普遍致命疾病的患者带来新治疗选择的重要性，”Klotho首席执行官约瑟夫·辛库勒博士表示，“我的表亲凯伦死于这种可怕的疾病。”

We aim to deliver the first gene replacement therapy addressing the neurologic insult resulting in motor neuron damage and the potential neurologic protection induced by providing therapeutic blood, brain, and muscle concentrations of the s-KL protein. After the FDA's review of the data leading to the Orphan Drug Designation, we believe this ODD designation provides strong validation of our science and our approach to treat this disease' concludes Dr.

我们的目标是提供首个基因替代疗法，解决导致运动神经元损伤的神经损伤问题，并通过提供治疗性血液、大脑和肌肉浓度的s-KL蛋白，实现潜在的神经保护。在FDA审查了导致孤儿药资格认定的数据后，我们相信这一ODD资格认定为我们的科学和治疗该疾病的方案提供了强有力的验证。" 博士总结道。

Sinkule..

辛库勒..

ALS is sometimes referred to as

ALS 有时被称为

Lou Gehrig's

卢·格里克病

disease.

疾病。

Lou Gehrig

卢·格里克

, who played for the New York Yankees for 17 years in the 1920s and 1930s, stunned players and fans by retiring from baseball at the age of 36 after being diagnosed with ALS. Prior to this diagnosis, Gehrig played in a record-breaking 2,130 consecutive games, was referred to as the 'Iron Horse,' and was considered one of the greatest baseball players of all time.

，他在1920年代和1930年代为纽约洋基队效力了17年，在被诊断出患有ALS后，于36岁时宣布退役，震惊了球员和球迷。在确诊之前，格赫里格连续参加了创纪录的2,130场比赛，被称为“铁马”，并被认为是有史以来最伟大的棒球运动员之一。

Less than two years later, at the age of 37, Gehrig died of complications from ALS. ALS is also referred to as Motor Neuron Disease in the UK and elsewhere. ALS is considered a rare disease and affects fewer than 200,000 people in the US, with around 5,000 new cases diagnosed each year..

不到两年后，37岁的格里克因ALS并发症去世。ALS在英国和其他地方也被称为运动神经元病。ALS被认为是一种罕见疾病，在美国影响不到20万人，每年大约有5000例新病例被诊断出来。

Klotho Neurosciences will have completed 'proof of concept' studies in two animal models of human ALS and the Company is currently initiating manufacturing of the ALS-targeted product candidate, followed by meetings with the U.S. FDA and EMA in

克洛托神经科学公司将在两种人类ALS动物模型中完成“概念验证”研究，该公司目前正开始生产针对ALS的产品候选药物，并随后与美国FDA和EMA举行会议。

Europe

欧洲

to concur with the development path going forward.

同意未来的发展路径。

KLTO-202, the company's lead product candidate targeting motor neuron diseases and muscular dystrophies, is composed of a muscle-specific promoter called 'desmin,' driving the expression of the s-KL gene transcript and s-KL protein, with targeted delivery of the gene therapy to the neuromuscular junction - the interface between the spinal cord and the muscles.

KLTO-202 是该公司针对运动神经元疾病和肌肉萎缩症的主要候选产品，它由一个名为“结蛋白”的肌肉特异性启动子组成，驱动 s-KL 基因转录本和 s-KL 蛋白的表达，并通过基因疗法靶向递送到神经肌肉接头——即脊髓与肌肉之间的界面。

KLTO-202 is not approved for human use by any regulatory authority.

KLTO-202 未获任何监管机构批准用于人体。