China-based gene therapy start-up Accuredit Therapeutics (Suzhou) Co., Ltd has announced the first dosing of its investigational drug ART001 in seven subjects with transthyretin amyloidosis (ATTR), as part of an investigator-initiated trial (ITT). The drug candidate has demonstrated good safety and achieved a clinically significant decrease in transthyroxine (TTR) protein levels within two weeks of administration.
ATTR and Its Impact
ATTR is a serious, progressive, and life-threatening disease caused by the misfolding of TTR proteins, primarily originating from the liver, leading to the abnormal deposition of amyloid in tissues. The condition was recently included in China’s second batch rare disease list, highlighting the urgent need for effective treatments.
ART001: Non-Viral Vector Gene Editing Therapy
ART001 is described as the first non-viral vector-based in vivo gene editing drug to have entered human clinical trials in China. The therapy utilizes lipid nanoparticles (LNPs) as delivery carriers to safely and specifically edit TTR genes in the liver, blocking the expression of TTR proteins and preventing the abnormal deposition of amyloid substances.-Fineline Info & Tech
