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AccurEdit Therapeutics

Ownership: Private
Employees: ~10
Development stage: Phase 1

Overview

AccurEdit’s lead product ART001 is the first LNP-delivered in vivo gene editing therapy to enter human clinical trials in China and the U.S. It has shown >90% serum TTR reduction after a single dose with no significant safety issues or off-target effects observed over 15+ months of follow-up. ART002 achieved pharmacodynamic saturation for PCSK9 knockdown and LDL-C reduction (>50%, up to ~70%) with an excellent safety profile. Both programs are considered best-in-class globally for their respective indications[6][7][9].

Therapeutic areas: CardiovascularRare Diseases
Modalities: In vivo gene editingCRISPR-based therapiesLipid nanoparticle (LNP)-delivered gene editingBase editors (e.g., ARTbase-A1™)
Industry: Biotech

Drug pipeline

5 assets

ARTbase-A1

Preclinical

4 more assets in the full pipeline.

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Partnerships

No major pharmaceutical partnerships disclosed as of May 2025; collaborations primarily with academic hospitals for investigator-led trials [4][9].

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