By Fineline Cube 
China-based gene editing therapy specialist AccurEdit Therapeutics (Suzhou) Co., Ltd. has published the latest data for its drug ART001, a treatment for transthyretin amyloidosis (ATTR). ART001 is the first non-viral vector in vivo gene-edited drug in China to enter an investigator-initiated trial (IIT) and has demonstrated excellent safety and efficacy through 72-week clinical observations.
Clinical Trial Results
As of now, all participants have completed a 72-week follow-up. The latest clinical data show that after 72 weeks of administration, the high-dose group exhibited an average reduction in peripheral TTR protein levels compared to baseline, with reductions exceeding 90% and individual cases reaching up to 95%. These results highlight the drug’s potential to effectively manage ATTR.
Safety Profile
In terms of safety, no delayed adverse events were observed, and no new clinically significant safety concerns emerged. The drug’s favorable safety profile supports its continued development and potential use in treating transthyretin amyloidosis.-Fineline Info & Tech