SUZHOU, China, April 26, 2024 /PRNewswire/ -- Ractigen Therapeutics, a leader in the development of small activating RNA (saRNA) therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application for RAG-01, a groundbreaking saRNA therapy targeting non-muscle invasive bladder cancer (NMIBC).

中国苏州，2024年4月26日/PRNewswire/--RacGen Therapeutics是小激活RNA（saRNA）疗法开发的领导者，今天宣布，美国食品和药物管理局（FDA）已批准该公司针对RAG-01的研究性新药（IND）申请，RAG-01是一种针对非肌肉浸润性膀胱癌（NMIBC）的开创性saRNA疗法。

This approval facilitates the launch of U.S. clinical trials, following the successful initiation of a Phase I trial in Australia..

在澳大利亚成功启动I期试验后，该批准有助于启动美国临床试验。。

RAG-01 is a pioneering therapy in bladder cancer treatment, representing the first of its kind to specifically target and activate the p21 tumor suppressor gene. This gene is critical in regulating cell cycle progression and is a key component in stopping the growth of cancer cells. By activating p21, RAG-01 offers a targeted approach to potentially curb the progression of NMIBC, a prevalent form of bladder cancer..

RAG-01是膀胱癌治疗的先驱疗法，代表了第一种特异性靶向和激活p21肿瘤抑制基因的疗法。该基因在调节细胞周期进程中至关重要，并且是阻止癌细胞生长的关键成分。通过激活p21，RAG-01提供了一种有针对性的方法，可以潜在地抑制NMIBC（一种普遍的膀胱癌形式）的进展。。

'FDA IND approval for RAG-01 is a major achievement for Ractigen and a significant advancement for saRNA technology worldwide,' said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. 'This first-in-class saRNA therapy harnesses the power of RNAa to target the p21 gene, offering a promising new option for patients with limited treatment choices.

RacGen Therapeutics创始人兼首席执行官龙成利博士说，FDA IND批准RAG-01是RacGen的一项重大成就，也是全球saRNA技术的重大进步这种一流的saRNA疗法利用RNAa的力量靶向p21基因，为治疗选择有限的患者提供了一种有前途的新选择。

This approval validates the potential of RAG-01 as a leading saRNA therapy and strengthens our position as innovators in RNA-based treatments.'.

该批准验证了RAG-01作为领先的saRNA疗法的潜力，并加强了我们在基于RNA的治疗中作为创新者的地位。”。

About RAG-01: RAG-01 is a pioneering saRNA candidate engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNAa. Traditionally considered 'undruggable,' p21 presents a unique opportunity for saRNA-based targeted activation. The drug, delivered through intravesical instillation using Ractigen's proprietary LiCO™ delivery technology, has shown significant tumor suppression in mouse orthotopic bladder cancer models.

关于RAG-01：RAG-01是一种开创性的saRNA候选物，旨在通过RNAa的机制靶向和激活肿瘤抑制基因p21。传统上被认为是“不可治愈的”，p21为基于saRNA的靶向激活提供了独特的机会。该药物使用Ractigen专有的LiCO™递送技术通过膀胱内滴注递送，在小鼠原位膀胱癌模型中显示出显着的肿瘤抑制作用。

Currently, the Phase I clinical trial of RAG-01 in Australia has successfully enrolled and dosed the first three patients. Its development marks a significant stride in RNAa based therapies, addressing the unmet needs of NMIBC patients..

目前，RAG-01在澳大利亚的I期临床试验已成功招募并给前三名患者服用。它的发展标志着基于RNAa的疗法迈出了重要的一步，解决了NMIBC患者未满足的需求。。

About NMIBC: NMIBC represents 50-80% of all bladder cancer cases. Despite standard treatments like transurethral resection of bladder tumor (TURBT) followed by intravesical BCG or chemotherapy, recurrence rates remain high, estimated at 50-70% within the first five years. RAG-01's development is a significant step towards addressing this substantial unmet need in bladder cancer therapy..

关于NMIBC：NMIBC占所有膀胱癌病例的50-80%。尽管有标准治疗方法，如经尿道膀胱肿瘤切除术（TURBT），然后进行膀胱内卡介苗或化疗，但复发率仍然很高，估计在前五年内复发率为50-70%。RAG-01的开发是解决膀胱癌治疗中这一尚未满足的巨大需求的重要一步。。

About LiCO™: LiCO™, Ractigen's proprietary extra-hepatic delivery system, enables the delivery of duplex RNA into a variety of tissues and organs which are hard to reach by conventional approach. It supports multiple administration routes, including subcutaneous, intravenous, intravesical, and intravitreal, providing profound and durable activity.

关于LiCO™：LiCO™，Ractigen专有的肝外递送系统，能够将双链RNA递送到常规方法难以到达的各种组织和器官中。它支持多种给药途径，包括皮下，静脉内，膀胱内和玻璃体内，提供深刻而持久的活动。

Its versatility and effectiveness have been validated in many preclinical studies, marking it as a cornerstone in Ractigen's therapeutic arsenal..

它的多功能性和有效性已在许多临床前研究中得到验证，标志着它是Racigen治疗武器库的基石。。

About RNAa: Pioneered by Dr. Long-Cheng Li and his team, RNAa is a clinically validated platform technology. It employs saRNA to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This technology has vast potential for developing therapeutic drugs across various diseases, especially where traditional methods fall short, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders..

关于RNAa：由李龙成博士和他的团队开创，RNAa是一种经过临床验证的平台技术。它利用saRNA靶向基因调控域，激活基因表达并恢复治疗性蛋白质水平。这项技术在开发各种疾病的治疗药物方面具有巨大潜力，特别是在传统方法不足的情况下，包括癌症，遗传疾病，慢性疾病以及代谢和脑血管疾病。。

About Ractigen Therapeutics: A leader in saRNA drug development, Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing the RNAa mechanism to up-regulate endogenous gene expression. This innovative approach involves saRNA targeting specific genes to enhance transcription, thereby restoring normal protein functions.

关于Racigen Therapeutics：Racigen Therapeutics是saRNA药物开发的领导者，它处于开发利用RNAa机制上调内源基因表达的saRNA药物的前沿。这种创新方法涉及靶向特定基因的saRNA以增强转录，从而恢复正常的蛋白质功能。

Ractigen's cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation. .

Ractigen的尖端技术对于治疗常规方法无法解决的疾病至关重要，例如表观遗传沉默或基因下调导致的疾病。。

Contact Information:

联系方式：

Ractigen Therapeutics

Ractigen治疗学

www.ractigen.com

www.ractigen.com

View original content:https://www.prnewswire.com/news-releases/ractigen-therapeutics-announces-fda-approval-for-rag-01-a-first-in-class-sarna-therapy-for-bcg-unresponsive-nmibc-302128680.html

查看原始内容：https://www.prnewswire.com/news-releases/ractigen-therapeutics-announces-fda-approval-for-rag-01-a-first-in-class-sarna-therapy-for-bcg-unresponsive-nmibc-302128680.html

SOURCE Ractigen Therapeutics

来源：Ractigen Therapeutics