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RMAT designation expected to expedite development; 72 weeks interim clinical trial data showing sustainable reduction of TTR over 90% with no significant adverse events.
SUZHOU, China, May 29, 2025 /PRNewswire/ — AccurEdit Therapeutics today announced that its internally developed in vivo gene editing therapy ART001 has been granted a Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA). Public records indicate that ART001 is the first gene editing product from China to receive an FDA RMAT designation, highlighting the FDA’s recognition of its innovative nature and clinical potential. This designation is expected to help expedite the clinical development of ART001 for patients with ATTR amyloidosis.
Based on the existing clinical data, the FDA confirmed that ART001 meets the criteria for RMAT designation. Clinical results have shown that four weeks after administration, subjects in the high-dose cohort experienced an average >90% reduction in peripheral TTR protein levels from baseline, with this reduction sustained for 72 weeks to date.
ART001 has also exhibited a strong safety profile in clinical studies. In an investigator-initiated trial that has been followed up for 72 weeks by far, no infusion-related reactions or AST (aspartate aminotransferase) elevations—side effects commonly observed with similar therapies—were reported. Importantly, no dose-limiting toxicities (DLTs) or serious adverse events (SAEs) occurred during the study. ART001 demonstrated exceptional precision in pre-clinical studies, with no off-target gene edits detected even at dose levels many times higher than the therapeutic dose. These safety and efficacy findings support ART001’s potential as a globally leading, best-in-class treatment for ATTR.
ART001 has achieved several milestones in its development. In August 2023, ART001 became the first lipid nanoparticle (LNP)-delivered in vivo gene editing drug to enter a human clinical trial in China. It later received clearance to conduct clinical trials in both China and the United States (China in July 2024 and U.S. in August 2024), making it the only therapy of its class with regulatory approval to initiate trials in both countries. ART001 has since advanced into a Phase IIa study. In March 2025, the FDA also granted ART001 Orphan Drug designation for the treatment of ATTR amyloidosis, further supporting its potential to address this life-threatening disease.
About Regenerative Medicine Advanced Therapy (RMAT) Designation
The RMAT designation was established under the U.S. 21st Century Cures Act of 2016 as a program to expedite the development and review of innovative regenerative medicine products, including cell therapies, gene therapies, tissue-engineered products, and combination products. An investigational therapy may receive RMAT designation if it is intended to treat a serious or life-threatening condition and preliminary clinical evidence indicates the therapy has the potential to address unmet medical needs by demonstrating a meaningful therapeutic effect on the disease.
About AccurEdit Therapeutics
AccurEdit Therapeutics, founded in 2021, is a biotechnology company focused on developing in vivo gene editing technologies and therapies based on lipid nanoparticle (LNP) and other non-viral delivery systems. The company is dedicated to providing one-time, cost-effective, innovative treatment solutions to patients worldwide. It has assembled a specialized team with successful experience across the biologic drug development lifecycle and has established the world’s first clinically validated, industrial-scale, end-to-end in vivo gene editing platform. AccurEdit Therapeutics has built a broad intellectual property portfolio covering its technologies and products, including a novel base editor ARTbase-A1™, which has been granted a U.S. patent.
AccurEdit Therapeutics is advancing a pipeline targeting both rare genetic diseases and difficult-to-treat common diseases. In August 2023, its lead product ART001 (for ATTR amyloidosis) became the first LNP-delivered in vivo gene editing therapy to enter a human clinical trial in China. In August 2024, ART001 also became the first – and currently only – such product to receive an Investigational New Drug (IND) approval from the U.S. FDA. In February 2025, the company’s second product ART002 (targeting heterozygous familial hypercholesterolemia [HeFH] via the PCSK9 gene) became the world’s first in vivo gene editing product of its class to achieve pharmacodynamic saturation in humans and effectively reduce LDL-C levels in patients with extremely high baseline cholesterol. The safety and efficacy data from both ART001 and ART002 suggest these candidates have the potential to become best-in-class gene editing therapies globally.
