AccurEdit's ART001: First Gene Editing Therapy in China with FDA RMAT Designation

AccurEdit Therapeutics, a biotechnology company based in Suzhou, China, has achieved a significant milestone with its in vivo gene editing therapy, ART001, which has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA). This marks the first time a gene editing product from China has received such recognition from the FDA, underscoring the innovative and potentially transformative nature of ART001. The RMAT designation is expected to streamline the development process of ART001 for patients suffering from ATTR amyloidosis, a serious genetic condition.

ART001 has demonstrated remarkable efficacy in clinical trials, with data showing an average reduction of over 90% in peripheral TTR protein levels four weeks post-administration, a result maintained for up to 72 weeks. The therapy has also proven to be safe, with no infusion-related reactions or significant side effects such as AST elevations, which are common with similar treatments. Furthermore, no dose-limiting toxicities or serious adverse events were observed during the studies. Pre-clinical trials confirmed the precision of ART001, with no off-target gene edits detected even at doses much higher than the therapeutic level. These results position ART001 as a potential leading treatment worldwide for ATTR.

Since its inception, ART001 has reached several critical milestones. In August 2023, it became the first in vivo gene editing drug delivered via lipid nanoparticle (LNP) technology to enter human clinical trials in China. The therapy later received approval to conduct trials in both China and the United States, with regulatory clearances obtained in July 2024 and August 2024, respectively. ART001 has progressed into Phase IIa studies, and in March 2025, it was also granted Orphan Drug designation by the FDA for the treatment of ATTR amyloidosis, further reinforcing its potential to address this life-threatening condition.

The RMAT designation was created under the U.S. 21st Century Cures Act of 2016 to accelerate the development and review of cutting-edge regenerative medicine products. It applies to therapies intended to treat serious or life-threatening conditions, provided there is preliminary clinical evidence suggesting a meaningful therapeutic impact and the potential to address unmet medical needs.

Founded in 2021, AccurEdit Therapeutics is committed to developing innovative in vivo gene editing technologies using non-viral delivery systems like LNPs. The company's mission is to offer one-time, cost-effective treatment solutions to patients globally. With a team experienced in the biologic drug development lifecycle, AccurEdit has established a clinically validated, industrial-scale platform for in vivo gene editing, supported by a comprehensive intellectual property portfolio that includes a novel base editor, ARTbase-A1™, patented in the U.S.

In addition to ART001, AccurEdit is advancing a pipeline aimed at both rare genetic diseases and challenging common diseases. ART002, targeting heterozygous familial hypercholesterolemia (HeFH) via the PCSK9 gene, became the first in vivo gene editing product of its type to achieve pharmacodynamic saturation in humans, effectively reducing LDL-C levels in patients with high cholesterol. The data from ART001 and ART002 indicate their potential to become leading gene editing therapies on a global scale.