AccurEdit Therapeutics Co., Ltd. ("AccurEdit Therapeutics") announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for ART001, intended for the treatment of transthyretin (ATTR) amyloidosis with polyneuropathy.

ART001, China’s first LNP-based in vivo gene editing product, entered human clinical study (IIT) in 2023. Results from the study demonstrated that four weeks post-administration, subjects in the high-dose group experienced an average reduction of over 90% in peripheral TTR protein levels compared to baseline. Further follow-ups revealed that 36 weeks post-administration, the TTR reduction level remained stable.

ART001 had previously received NMPA’s IND approval in July 2024 in China. With FDA’s clearance today, ART001 becomes the first and only LNP-based in vivo gene editing product that cleared INDs in both China and the US.