China-based AccurEdit Therapeutics (Suzhou) Co., Ltd announced receiving Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its investigational drug ART001, targeting transthyretin amyloidosis (ATTR). This marks a significant milestone in the development of this innovative gene-editing therapy.
Drug Profile and Mechanism
ART001 is the first non-viral vector in vivo gene-edited drug to receive clinical approval in both the US and China. It is also the first of its kind in China to enter investigator-initiated trials (ITTs). The drug demonstrates a unique mechanism of action, achieving over 90% TTR knockdown with a single use, indicating industry-leading effectiveness.
Clinical Data and Safety
Clinical data shows that ART001’s efficacy has been consistently maintained for at least 15 months, surpassing the normal renewal and regeneration cycle of the human liver. This provides direct clinical evidence that ART001 may only require a single lifetime administration. In IIT and regulatory Phase I/IIa clinical studies, ART001 exhibited excellent off-target safety with no infusion-related reactions, even at doses several tens of times the saturation level.-Fineline Info & Tech
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