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.
“FDA clearance of our first IND is a significant milestone for our company. It validates the ability for our Targeted AID-mediated Mutagenesis (TAM) cytosine base editor technology to target diseases previously considered untreatable—broadening the full-body application of gene editing technology. As the second generation of CRISPR-Cas9, base editors offer immense potential with significantly lower off-target risks.” said Dr. Chunyan He, CEO of GenAssist, “GenAssist is the first company to apply gene editing drug for DMD indication. [Read More: What is Exon Skipping and How does it Work?]
By permanently repairing the mutated DMD gene, base editing may provide long-term benefit for DMD patients. We are excited about the potential of this program to bring new treatment for patients and demonstrate gene editing as a new therapeutic approach for DMD. In addition, GenAssist is advancing other DMD exon-skipping programs, which cover more than 30% DMD population.”
