ART001 / AccurEdit Therapeutics - LARVOL DELTA

ART001: Development and Interim Clinical Outcomes of a CRISPR-Based In Vivo Gene-Editing Therapy for Hereditary ATTR (ASGCT 2025) - "With regulatory approvals and promising, 48-weeks, interim clinical results, ART001 holds the potential to revolutionize ATTR treatment, reduce treatment burden, and significantly improve patient outcomes. Disease Focus of Abstract:Genetic Diseases"

Clinical data • Preclinical • Amyloidosis • Dyslipidemia • Genetic Disorders • Hepatology • Hypertriglyceridemia • Musculoskeletal Pain • Pain

AccurEdit Therapeutics’ LNP-based In Vivo Gene Editing Product ART001 Obtained Orphan Drug Designation from the FDA (PRNewswire) - "AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). ART001, which utilizes lipid nanoparticle (LNP) technology for targeted gene editing, is designed for the treatment of transthyretin amyloidosis (ATTR), a rare and life-threatening disease."

Orphan drug • Amyloidosis