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

publication date: May 10, 2023

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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