GEN6050

**GEN6050** is an investigational dual-AAV9 in vivo base-editing gene therapy developed by GenAssist Therapeutics for **Duchenne muscular dystrophy** in patients whose mutations are amenable to dystrophin exon 50 skipping. It delivers an optimized Targeted AID-mediated Mutagenesis cytosine base editor and an exon 50 guide RNA to edit the intron 50 5′ splice-site region of the human **DMD** gene, driving exon 50 skipping at the mRNA level and enabling production of a truncated, in-frame dystrophin protein. The reported preclinical program used two AAV9 vectors, ss.AAV9.oTAM and ss.AAV9.hE50-sgRNA. ([mdaconference.org](https://www.mdaconference.org/abstract-library/a-transformative-base-editing-drug-for-dmd-therapy/))