SUZHOU—AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading Chinese specialist in gene editing therapies, has announced that it has received approval from the US Food and Drug Administration (FDA) for its investigational drug ART001, which targets transthyretin amyloidosis. This milestone marks ART001 as the first in vivo gene-edited drug based on non-viral vectors from China to be approved for clinical trials in the US.
ART001 is currently undergoing a Phase I clinical study in China as the first non-viral vector in vivo gene-edited drug to enter investigator initiated trials (ITTs). The drug candidate has demonstrated a favorable safety profile, with no infusion-related reactions observed in subjects administered ART001, a stark contrast to other similar products that often exhibit medication reactions such as fever.
In the critical area of off-target editing safety, a major concern for gene-editing products, ART001 has shown no off-target editing at several tens of times the saturation dose. This achievement places AccurEdit among an elite group of only three companies globally that have obtained clinical approvals in the US for this class of drugs.
The FDA’s approval of ART001 for clinical trials in the US is a significant step forward for AccurEdit Therapeutics, showcasing the company’s commitment to advancing gene editing therapies and its potential to impact the global landscape of genetic medicine. – Flcube.com
