ABOUT US
OVERVIEW
MILESTONES
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SCIENCE & TECHNOLOGY PLATFORM
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PRESS RELEASE
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HOME
ABOUT US
OVERVIEW
MILESTONES
LEADERSHIP
INVESTORS
SCIENCE & TECHNOLOGY PLATFORM
PIPELINE
PRESS RELEASE
CONTACT US
Accurate Editing for Cures
Delivering the best in vivo gene editing medicine
Mar 20, 2025
AccurEdit Therapeutics' LNP-based In Vivo Gene Editing Product ART001 Obtained Orphan Drug Designation from the FDA
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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
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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
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> 90% serum TTR reduction after 4 weeks
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No off target editing detected at up to ~80 times of EC90
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No infusion-related reaction observed
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No > Grade 1 liver enzyme elevation observed
PLATFORM THAT PERFORMS
Industry-leading end-to-end in vivo gene editing platform
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RNA synthesis (Research and GMP grade)
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Editing (Patented base editors and Cas enzymes; industry-leading capability of gRNA screening and optimization)
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Delivery (Industry-leading efficiency and specificity)
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