GenAssist Ltd (GenAssist), announced the first DMD patient dosed with its base editing drug, GEN6050X injection, in an investigator-initiated trial (IIT).

"This is the first-in-human trial for DMD gene editing therapy. It marks that DMD treatment has entered the gene editing era", said Chelsie He, CEO of GenAssist, "As the second generation of CRISPR-Cas9, base editors own huge application potential with lower off-target risk. DMD is such a devastating disease and lack effective drugs. DMD families suffer a lot from this disease. Leveraging this groundbreaking technology, we are dedicated to developing better treatments for DMD population."

 GEN6050X Injection

GEN6050X injection is an intravenous cytosine base editing drug using dual AAV9 vectors, designed for DMD patients amenable to exon 50 skipping. GEN6050X is based on GenAssist's unique RNA editing-free Targeted AID-mediated Mutagenesis (TAM) cytosine base editor technology. Through one-shot systemic administration, GEN6050X can permanently restore the expression of dystrophin through editing the mutated DMD gene. It may provide a curative solution for DMD. The IIT study is being conducted in the Peking Union Medical College Hospital (NCT06392724). At the same time, the IND filing of GEN6050X (US and China) is expected in November 2024.