SUZHOU, China, May 15, 2024 /PRNewswire/ -- Ractigen Therapeutics, a clinical-stage pharmaceutical company committed to developing pioneering therapies, today announces that the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) has approved the Investigational New Drug (IND) application for the initiation of Phase 1 clinical trials in China for RAG-17, targeting Amyotrophic Lateral Sclerosis (ALS)..

中国苏州，2024年5月15日/PRNewswire/--致力于开发开创性疗法的临床阶段制药公司Ractigen Therapeutics今天宣布，中国国家医药产品管理局（NMPA）药物评估中心（CDE）已批准研究性新药（IND）申请，在中国启动针对肌萎缩侧索硬化症（ALS）的RAG-17的1期临床试验。。

The IND-opening study is a Phase I, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of RAG-17 in subjects with ALS carrying the Superoxide Dismutase 1 (SOD1) mutation.

IND开放研究是一项I期随机双盲安慰剂对照研究，评估RAG-17在携带超氧化物歧化酶1（SOD1）突变的ALS患者中的安全性，耐受性，药代动力学（PK）和药效学（PD）。

Dr. Long-Cheng Li, Founder, President, and CEO of Ractigen, expressed enthusiasm about this significant milestone: 'This marks a pivotal moment for our company, as RAG-17 is our first siRNA drug targeting the central nervous system to receive CDE approval. We are excited to advance this therapy into clinical trials for ALS patients and are optimistic about its potential to bring meaningful benefits to those with SOD1 mutation.'.

Ractigen的创始人、总裁兼首席执行官龙成利博士对这一重要里程碑表示了热情：“这标志着我们公司的关键时刻，因为RAG-17是我们第一个获得CDE批准的针对中枢神经系统的siRNA药物。我们很高兴将这种疗法推进ALS患者的临床试验，并对其为SOD1突变患者带来有意义益处的潜力感到乐观。”。

Mr. Lei Cai, a prominent ALS advocate and fighter against the disease, who himself battles ALS in China, shared his hopeful perspective on RAG-17: 'I hold a strong sense of optimism for RAG-17. Its innovative approach and promising preliminary results give me great confidence in its potential. I believe this drug could offer significant hope and tangible benefits to the ALS community, especially for those with the SOD1 mutation.'.

著名的肌萎缩侧索硬化症倡导者和抗击该病的斗士蔡磊（音译）在中国与肌萎缩侧索硬化症作斗争，他分享了他对RAG-17的乐观观点：“我对RAG-17抱有强烈的乐观情绪。它的创新方法和有希望的初步结果使我对其潜力充满信心。我相信这种药物可以为ALS社区提供巨大的希望和切实的好处，特别是对于那些SOD1突变的人。”。

RAG-17 previously received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in March 2023 and has been cleared for clinical trials in the U.S. Additionally, an Investigator-Initiated Trial (IIT) study (NCT05903690) for RAG-17 is currently ongoing, with 6 patients recruited and treated, showing encouraging preliminary safety and efficacy results..

RAG-17之前于2023年3月从美国食品和药物管理局（FDA）获得孤儿药指定，并已被批准在美国进行临床试验。此外，RAG-17的研究者发起试验（IIT）研究（NCT05903690）目前正在进行中，招募并治疗了6名患者，显示出令人鼓舞的初步安全性和有效性结果。。

About RAG-17

关于RAG-17

RAG-17 is a therapeutic siRNA specifically designed to suppress the SOD1 gene in ALS patients with pathogenic mutations. Utilizing Ractigen's proprietary SCAD™ delivery platform, RAG-17 combines siRNA with an accessory oligonucleotide (ACO) for enhanced effectiveness in central nervous system (CNS) tissues.

RAG-17是一种治疗性siRNA，专门设计用于抑制具有致病突变的ALS患者的SOD1基因。RAG-17利用Ractigen专有的SCAD™递送平台，将siRNA与辅助寡核苷酸（ACO）结合，以增强中枢神经系统（CNS）组织的有效性。

Preclinical studies, including those using the hSOD1G93A mouse model, have demonstrated RAG-17's remarkable therapeutic efficacy in ameliorating motor function and prolonging survival. .

临床前研究，包括使用hSOD1G93A小鼠模型的研究，已经证明RAG-17在改善运动功能和延长生存期方面具有显着的治疗功效。。

About ALS

关于ALS

ALS, a severe neurodegenerative disease with no cure, significantly reduces life expectancy, with most patients succumbing to respiratory failure within 3-5 years of diagnosis. Initial symptoms typically include muscle cramps, twitching, and weakness. These symptoms progress to difficulties with movement and speech, the need for assisted breathing, paralysis, and ultimately death.

ALS是一种无法治愈的严重神经退行性疾病，可显着降低预期寿命，大多数患者在诊断后3-5年内死于呼吸衰竭。最初的症状通常包括肌肉痉挛，抽搐和虚弱。这些症状会发展为运动和言语困难，需要辅助呼吸，瘫痪，最终导致死亡。

Of the more than 50 genes associated to ALS, mutations in the SOD1 gene account for approximately 20% of the cases..

在与ALS相关的50多个基因中，SOD1基因突变约占病例的20%。。

About Ractigen Therapeutics

关于Ractigen Therapeutics

Ractigen Therapeutics, a clinical-stage pharmaceutical company, is committed to developing innovative oligonucleotide drugs and therapeutic approaches. Ractigen has pioneered several globally leading oligonucleotide drug delivery platforms, including SCAD™ and LiCO™, and boasts a highly diverse pipeline of oligonucleotide drugs.

Ractigen Therapeutics是一家临床阶段制药公司，致力于开发创新的寡核苷酸药物和治疗方法。Ractigen开创了几个全球领先的寡核苷酸药物输送平台，包括SCAD™和LiCO™等，并拥有高度多样化的寡核苷酸药物管道。

These drugs are aimed to target a wide variety of indications, from neurodegenerative and neuromuscular diseases to cancer, metabolic disorders, and hematological diseases. The company is dedicated to delivering innovative therapeutic solutions for previously undruggable targets and addressing incurable diseases across various fields.

这些药物旨在针对各种适应症，从神经退行性疾病和神经肌肉疾病到癌症，代谢紊乱和血液疾病。该公司致力于为以前无法治愈的目标提供创新的治疗方案，并解决各个领域的不治之症。