GEN6050 / GenAssist Therap - LARVOL DELTA

A clinical DMD cytosine base editing drug (ASGCT 2025) - P1 | "Collectively, GEN6050X is a safe and effective gene editing drug candidate and may provide a cure for DMD exon 50 skipping patients. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Clinical • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Hepatology

GenAssist Ltd Announces FDA Clearance of Investigational New Drug Application for GEN6050X, a Globally First-in-Class Base Editing Drug for Duchenne Muscular Dystrophy (PRNewswire) - "On March 06, 2025, GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in genome medicines, is thrilled to announce that it has received clearance from the U.S Food and Drug Administration(FDA) for its Investigational New Drug (IND) application for GEN6050X, a first-in-class base editing drug for Duchenne Muscular Dystrophy (DMD). GenAssist is planning to conduct clinical study globally for GEN6050X."

IND • Duchenne Muscular Dystrophy

GEN6050XIIT: A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (clinicaltrials.gov) - P1 | N=3 | Recruiting | Sponsor: Peking Union Medical College Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

GEN6050XIIT: A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (clinicaltrials.gov) - P1 | N=3 | Not yet recruiting | Sponsor: Peking Union Medical College Hospital

Gene therapy • New P1 trial • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Potency and Off-Target Evaluation of Targeted AID-Mediated Mutagenesis (TAM) Base Editor In Vitro (ASGCT 2024) - "The editing window of GEN6050 was defined as 10 bp upstream to 30 bp downstream of hE50-sgRNA (from +10 to -30 of hE50-sgRNA) corresponding to -24 to +36 bp of dystrophin gene IVS50 5'SS according to the efficient editing of GEN6050 (MOI=3E5) in normal iPSCs derived myotubes. Dose dependent target site editing, and exon 50 skipping were observed. The exon skipping efficiency is highly correlated with the editing efficiency, confirmed that the exon skipping is caused by efficient DNA modification at IVS50 5'SS."

Preclinical • Oncology

A Transformative Base Editing Drug for DMD Therapy (MDA 2024) - "Conclusion In summary, GEN6050 leverages base editing activities to restore the Dystrophin protein in DMD iPSC derived cardiomyocytes/myotubes with low to non-detectable off-target risk. Our results indicate that GEN6050 can provide a potential cure for DMD exon 50 skipping amenable patients."

Duchenne Muscular Dystrophy • Genetic Disorders

Suzhou GenAssist Therapeutic Co.,Ltd recently announced that its first base editing product, GEN6050, has submitted a pre-IND application to the FDA and has been accepted (PRNewswire) - "Suzhou GenAssist Therapeutic Co.,Ltd recently announced its pre-IND application of their first base editing product, GEN6050 and the acceptance by the FDA. GEN6050 is an in vivo base editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene. It is designed to restore the expression of dystrophin protein by base editing...The Pre-IND meeting usually refers to a voluntary meeting where the sponsor submits and communicates with the regulatory authority before conducting clinical trials of investigational new drugs (IND). The main purpose of the meeting is to review pharmacological study and non-clinical study, and to discuss whether existing research results are sufficient to support clinical research."

FDA event • Duchenne Muscular Dystrophy