On September 06, 2024, GenAssist Ltd (GenAssist), announced that the first DMD patient has been successfully dosed with its base editing drug, GEN6050X injection, in an investigator-initiated trial (IIT).
“To date, no gene editing drug has entered clinical trials for DMD indication. This first-in-human dosing 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. With the permanent recovery of mutated DMD gene, base editing enables lower dose to obtain long-term benefit compared to gene replacement therapy. The IIT study will facilitate us to accumulate more experience about this innovative technology. So far, the patient has already been discharged on schedule and has completed the 30-day follow up. 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 more DMD patients.”
About 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.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a rare, X-linked recessive hereditary disease caused by mutations in the dystrophin gene, leading to a loss of dystrophin protein vital for muscle cell membrane stability. DMD primarily affects skeletal and heart muscles, with progressive muscle wasting symptoms leading to loss of ambulation by around age 12, followed by further complications including heart and respiratory failure, with an average life expectancy of 26 years.
DMD affects approximately 1 in 3,500 to 5,000 live male births. According to LEIDEN data, about 80% of DMD patients could potentially benefit from exon skipping, with 4% specifically eligible for exon 50 skipping.
