AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading specialist in gene editing therapies based in China, has received approval from the Center for Drug Evaluation (CDE) for its investigational drug ART001. This drug is designed to target transthyroxine protein amyloidosis, a severe genetic disorder. ART001 marks a significant advancement in China’s gene editing landscape as the first non-viral vector in vivo gene-edited drug to enter an investigator-initiated trial (ITT).
In the ITT initiated in August 2023, ART001 demonstrated promising results, showing a decrease of over 90% in transthyretin (TTR) levels from baseline after just four weeks of administration. This effect was found to be stable up to 24 weeks, with no off-target editing observed. The successful outcomes of the ITT have paved the way for AccurEdit’s plans to submit a clinical filing for ART001 in the US in the near future, potentially bringing this innovative therapy to a broader patient population.- Flcube.com
