AccurEdit Therapeutics (Suzhou) Co., Ltd. (“AccurEdit”) announced that its self-developed product ART001 targeting transthyretin amyloidosis (ATTR) has recently received IND clearance from the U.S. FDA, making it the first non-viral vector-based in vivo gene editing drug from China to obtain U.S. clinical trial authorization.
ART001 is China’s first non-viral vector-based in vivo gene editing drug to enter human clinical trials (IIT) and is currently undergoing Phase 1 clinical trials in China. With this U.S. FDA IND approval, ART001 has become the world’s only product of its kind to secure clinical trial authorizations from both China and the United States.
As a drug development partner of AccurEdit, VeloGene Biotechnology(MingCeler) extends its warmest congratulations on this exciting milestone and wishes AccurEdit continued success and breakthroughs in its future journey of innovative drug development. At the same time, MingCeler is immensely proud to have participated in the research and development of ART001. Utilizing our TurboMice™ high-efficiency tetraploid complementation technology, we rapidly completed the iteration of two generations of new target animal models for AccurEdit in just 4 months. We successfully developed humanized gene-editing mouse models carrying human genetic rare-disease mutations with ideal phenotypes and rapidly scaled up their production for ART001 preclinical studies, contributing to the acceleration of innovative gene therapy research and development for ATTR diseases.