ART001: China's First Non-Viral In Vivo Gene Editing Therapy Demonstrates Safety and Efficacy in IIT Clinical Trial for ATTR Amyloidosis

On October 20, 2023, the exploratory investigator-initiated trial (IIT) titled “Clinical Study of ART001 Injection for the Treatment of Transthyretin Amyloidosis (ATTR),” initiated by Suzhou Dushu Lake Hospital Affiliated to Soochow University and supported by RayzeGene (Suzhou) Co., Ltd. (hereinafter referred to as “RayzeGene”) and the Cord Rare Disease Center, successfully completed dosing in seven subjects. All patients demonstrated favorable safety profiles, with a clinically significant reduction in transthyretin (TTR) protein levels observed as early as two weeks post-dosing.

ART001 Injection is the first non-viral vector-based in vivo gene editing therapy in China to enter human clinical trials, demonstrating encouraging druggability.

ATTR is a severe, progressive, and life-threatening disease caused by the misfolding of transthyretin (TTR) protein, primarily produced by the liver, leading to abnormal deposition of amyloid substances in tissues. In September 2023, the National Health Commission included ATTR in the Second Batch of the Rare Disease Catalogue.

ART001 Injection is independently developed by RayzeGene based on its industry-leading, industrial-scale in vivo gene editing technology platform. Using lipid nanoparticles (LNPs) as the delivery vehicle, it delivers gene editing components targeting the TTR gene to the liver, safely and specifically editing the TTR gene to block TTR protein expression, thereby preventing the abnormal deposition of amyloid substances at their source. Regarding off-target editing, which is a primary concern for gene editing products, testing of ART001 Injection demonstrated that no off-target editing was detected even at doses more than 20 times the saturation dose. Its off-target safety profile comprehensively surpasses that of similar foreign products, placing it at the forefront of the industry.

Unlike small-molecule drugs, which require daily administration and can only slow disease progression, in vivo gene editing therapies have the potential to halt, and even reverse or cure, diseases with a single lifetime dose. RayGene’s in vivo gene technology platform does not rely on viruses or cells; consequently, the cost of its developed products will not reach the millions of RMB often associated with other gene and cell therapies, thereby generating substantial savings for both patients and the national medical insurance system.

Professor Hao Yonggang, Principal Investigator of the IIT Clinical Study on ART001 Injectionstated: “Currently, treatment options for patients with ATTR amyloidosis in China are extremely limited. The ART001 injection represents a milestone breakthrough in domestic drug development. Our existing data demonstrate that the drug exhibits outstanding safety and efficacy, and requires only a single dose, offering ATTR patients a novel and revolutionary therapeutic option while significantly alleviating the financial burden on both patients and the national healthcare insurance system. We will continue to advance the clinical research of ART001 injection, accumulate more clinical data, support indigenous innovative drugs, facilitate clinical translation, and provide better treatment options for more patients.”

Mr. Huang Rufang, Founder and Director of the Cord Disease Center for Rare DiseasesHe stated, “The most urgent need facing patients with rare diseases in China is the lack of medical resources and medications. For over a decade, my team and I have been promoting the introduction and development of drugs for rare diseases. We are particularly delighted to see that the RayzeGene team has developed a therapeutic drug for ATTR patients in just two years, leveraging globally leading in vivo gene editing technology. Notably, the data from the Investigator-Initiated Trial (IIT) have demonstrated excellent safety and potential efficacy. We look forward to advancing to the next phase of clinical trials as soon as possible!”

Dr. Wang Yongzhong, Founder, Chairman, and CEO of RuiZheng GeneHe stated, “We extend our special gratitude to Professor Hao and his team, the patients and families who participated in the project, the Cord Blood Rare Disease Center, and Director Huang for their support. We also sincerely thank all our colleagues at the company and other partners! RayZhen Gene is deeply honored to work alongside all these partners who care about patients, striving to develop a one-time treatment that provides lifelong benefits. It is truly encouraging that ART001 Injection has become China’s first in vivo gene editing product based on a non-viral vector to enter clinical trials in humans. However, larger-scale clinical trials are still needed in the future. We are fully aware that, as the developer of this product, we bear not only an industry responsibility but also the trust and confidence placed in us by patients, physicians, and all stakeholders. Leveraging the development experience of ART001 and the validation of our series of platform technologies, RayZhen Gene will accelerate the research and development of our pipeline products. We are committed to living up to this trust by creating more and better revolutionary in vivo gene editing therapies, thereby benefiting more patients at the earliest possible time.”

Suzhou Dushu Lake Hospital (The Affiliated Dushu Lake Hospital of Soochow University, Medical Center of Soochow University) is the first modern public hospital in Suzhou Industrial Park. As a directly affiliated hospital of Soochow University, it is a comprehensive medical institution integrating medical care, teaching, and scientific research. Leveraging the locational advantages of the Suzhou Area of the Jiangsu Free Trade Zone, the hospital adheres to its three core missions: delivering high-quality medical services, cultivating top-tier talent, and facilitating high-level biomedical translation. With a strategic vision for sustainable development, the hospital is committed to becoming a first-rate national public general hospital that is “research-oriented, internationalized, and high-level,” earning satisfaction from the government, confidence from the university, trust from the public, and respect from peers.

RuiZheng Gene (Suzhou) Co., Ltd. was established in July 2021, focusing on the development and commercialization of globally leading non-viral vector gene editing technologies and products. Leveraging a team with comprehensive experience across the entire lifecycle of biopharmaceuticals, RuiZheng Gene has built the only industrial-grade, end-to-end in vivo gene editing technology platform spanning China and the United States. It is the first innovative drug R&D company in China to advance non-viral vector-based in vivo gene editing therapeutics into human clinical trials.