Accurate Editing for Cures
Delivering the best in vivo gene editing medicine
Aug 29, 2024
AccurEdit Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application to Initiate a Phase One Trial of ART001 for the Treatment of Transthyretin (ATTR) Amyloidosis with Polyneuropathy
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Positively Transforming Patient's Life with Genome Editing
BREAKING THE GROUND FOR PATIENTS IN NEED
First in China: safe and effective systemic CRISPR gene editing in humans
●Proven capability and track record to deliver innovative products in China and the US in industry-leading speed
PRODUCT WITH AN EDGE
Potentially best-in-class in vivo gene editing product for ATTR
●> 90% serum TTR reduction after 4 weeks
●No off target editing detected at up to ~80 times of EC90
●No infusion-related reaction observed
●No > Grade 1 liver enzyme elevation observed
PLATFORM THAT PERFORMS
Industry-leading end-to-end in vivo gene editing platform
●RNA synthesis (Research and GMP grade)
●Editing (Patented base editors and Cas enzymes; industry-leading capability of gRNA screening and optimization)
●Delivery (Industry-leading efficiency and specificity)

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