AccurEdit Therapeutics 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.