AccurEdit Therapeutics(Suzhou) Co., Ltd. (referred to as "AccurEdit Therapeutics") recently announced that the U.S. IND application for its self-developed product ART001, targeting Transthyretin Amyloidosis (ATTR), has been approved. This makes ART001 the first non-viral vector-based in vivo gene editing drug in China to receive clinical trial authorization from the U.S. FDA.

 

ART001 is also the first non-viral vector-based in vivo gene editing drug in China to enter human clinical trials (IIT) and is currently undergoing Phase 1 clinical trials in China. With the FDA IND approval, ART001 has become the only product of its kind globally to receive clinical trial approvals from both China and the U.S.

 

As a partner in AccurEdit Therapeutics' drug development efforts, MingCeler Biotech extends warm congratulations on this milestone and wishes AccurEdit Therapeutics continued success and innovation in its future drug development journey. At the same time, MingCeler Biotech takes great pride in having participated in the R&D process of ART001. Utilizing its proprietary TurboMice™ high-efficiency tetraploid complementation technology, MingCeler Biotech rapidly completed two iterations of new target animal models for AccurEdit Therapeutics in just four months. The company developed and quickly scaled up production of humanized gene-edited mouse models carrying rare human genetic disease mutations with ideal phenotypes, which were used in preclinical studies of ART001, thereby contributing to accelerating the development of innovative gene therapies for ATTR.

 

About ATTR​

 

ATTR is a severe, progressive, and life-threatening disease caused by the misfolding of TTR protein (primarily produced in the liver) and the abnormal deposition of amyloid substances in tissues. 

 

Reference: AccurEdit Therapeutics Official WeChat Account