ART001 / AccurEdit Therapeutics - LARVOL DELTA

Ruizheng Gene's ART001 becomes China's first gene editing product to be recognized as an advanced regenerative medicine therapy by the FDA [Google translation] (Bioon.com) - "On May 21, 2025, Ruizheng Gene (Suzhou) Co., Ltd...announced today that its independently developed in vivo gene editing innovative drug ART001, which uses LNP as a carrier for the treatment of transthyretin-related amyloidosis (ATTR), has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (hereinafter referred to as FDA ). According to public information, this is the first gene editing product in China to be recognized by the U.S. FDA RMAT, which reflects the FDA's recognition of the innovation and clinical potential of ART001, and will strongly help ART001 accelerate clinical development."

FDA event • Amyloidosis

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 • 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