GenAssist Says US IND Accepted for Base Editing Product in Duchenne Muscular Dystrophy

Suzhou GenAssist Therapeutic reported the US FDA accepted an IND for its novel in vivo gene editing candidate as a treatment for Duchenne muscular dystrophy (DMD). GEN6050 is a base editing drug that is designed to restore the expression of dystrophin protein that is caused by exon 50 skipping in the DMD gene. GenAssist’s TAM BE (Targeted AID-based Mutagenesis) base editing technology was discovered by one of its Founders, Xing Chang, PhD, who now serves as the company’s CSO. DMD is a rare disease that occurs once per 4000 newborn boys. Exon 50 skipping can be used to treat 4% of DMD patients with large deletions. More details....

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