Cytosine base editing (Beam Therapeutics)

SUZHOU, China, Sept. 9, 2024 /PRNewswire/ -- On September 06, 2024, 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." 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. About GenAssist Ltd. Founded in July 2020, GenAssist Ltd. (GenAssist) is a pioneering gene editing drug company. The operation entity of GenAssist is located in Suzhou, China, with two subsidiaries in Shanghai (China) and Boston (US). GenAssist owns the global rights of Targeted AID-mediated Mutagenesis cytosine base editor technology. The company is dedicated to developing base editing drugs against life-threatening diseases. For more information, please visit , Contact: [email protected]. SOURCE GenAssist Therapeutic Co.,Ltd