Xinya Gene​ recently announced that the Investigational New Drug (IND) application for its first-in-class gene-editing drug ​GEN6050X, intended for the treatment of Duchenne Muscular Dystrophy (DMD), has been approved by the U.S. Food and Drug Administration (FDA).

With this FDA IND approval, ​GEN6050X injection has become the first gene-editing DMD candidate drug globally to enter clinical trials. Xinya Gene is now planning to conduct global clinical studies for GEN6050X.

Dr. He Chunyan, CEO of Xinya Gene, stated: “This is a major milestone for our company. The FDA’s approval of our first IND validates the capability of our Targeted AID-mediated Mutagenase (TAM) cytosine base editing technology, which can target previously untreatable diseases—thereby expanding the systemic application of gene editing technology. As a second-generation technology following CRISPR-Cas9, base editors offer significant therapeutic potential with markedly reduced off-target risks. Xinya is the first company globally to apply gene-editing therapeutics to DMD. By permanently repairing the mutated DMD gene, base editing technology may provide long-term benefits for DMD patients. We are excited about this project, as it not only offers a novel treatment approach for patients but also demonstrates that gene editing can serve as a new therapeutic strategy for DMD. In addition, Xinya is actively advancing other DMD exon-skipping projects, which are expected to cover over 30% of the DMD patient population.”

​MingCeler Biotech, leveraging its self-developed ​TurboMice™ technology, has developed multiple rare disease mouse models, such as GJB2-related deafness mice and transthyretin amyloidosis mouse models. The TurboMice™ technology overcomes challenges related to long modeling cycles and low success rates for intricate models, enabling precise editing at nearly any target gene locus. It allows for the generation of fully homozygous gene-edited mouse models directly from embryonic stem cells in as little as two months. Mingxun Biotech is currently focusing on the development of rare disease mouse models and offers rapid customization of various mouse models based on client needs. We welcome inquiries and collaborations!