CAMBRIDGE, Mass., March 25, 2025 /PRNewswire/ -- AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA).

ART001, which utilizes lipid nanoparticle (LNP) technology for targeted gene editing, is designed for the treatment of transthyretin amyloidosis (ATTR), a rare and life-threatening disease. It is currently the first and only LNP-based in vivo gene editing product worldwide that has cleared an IND in both China and the United States. It was also China's first LNP-based in vivo gene editing product to enter clinical study in human in August 2023. Clinical data shows that a single dose of ART001 can achieve a >90% reduction of transthyretin (TTR) in the serum. The level of reduction has remained stable for more than 15 months by now, exceeding the natural renewal cycle of the human liver. This is the first, and by far the only, evidence obtained in China to confirm the potential of in vivo gene editing therapy such as ART001 as a one-time, lifelong treatment.

Other in vivo gene editing products developed in the US and China have reported infusion-related reactions frequently in their clinical trials, including severe cases that require emergency care. No infusion-related reactions were observed in patients enrolled in the first clinical study of ART001 with at least 15 months follow up. Additionally, no off-target editing was detected even at doses tens of times higher than the saturating dose of ART001 in primary human hepatocytes.

Dr. Yongzhong Wang, Founder, Chairman, and CEO of AccurEdit Therapeutics, stated, "The orphan drug designation for ART001 by the FDA provides a solid foundation for its global development. Our team is excited to have the opportunity to develop ART001 as a safe, effective, and accessible treatment option for ATTR patients around the world."