By Fineline Cube 
China-based gene editing therapy specialist AccurEdit Therapeutics (Suzhou) Co., Ltd announced receiving Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its investigational drug ART001, targeting transthyretin amyloidosis (ATTR). This marks a significant milestone in the development of innovative treatments for this rare disease.
ART001 Development
ART001 is the first non-viral vector in vivo gene-edited drug in China to enter investigator-initiated trials (ITTs). It was approved for clinical trials in China and the US in July and August 2024, respectively. The drug, currently in Phase IIa trials, was awarded Orphan Drug Designation (ODD) in the US in March of this year.
Clinical Data and Safety Profile
Clinical data demonstrate that after 4 weeks of ART001 administration, the high-dose group achieved a mean reduction in peripheral TTR protein levels from baseline exceeding 90%, with sustained efficacy maintained for 72 weeks. Leveraging AccurEdit’s leading in vivo gene-editing platform, ART001 showed no infusion-related reactions or elevated aspartate aminotransferase (AST) levels—commonly observed in similar products globally—throughout its 72-week IIT. Additionally, no dose-limiting toxicities (DLT) or serious adverse events (SAEs) were reported. Notably, ART001 exhibited exceptional off-target safety, with no detectable off-target editing even at doses dozens of times above saturation levels.-Fineline Info & Tech