GenAssist Ltd Doses First DMD Patient with Base Editing Drug

SUZHOU, China, Sept. 9, 2024 – GenAssist Ltd (GenAssist) has announced a significant achievement in the field of gene therapy. On September 06, 2024, the company administered its pioneering base editing drug, GEN6050X injection, to the first Duchenne Muscular Dystrophy (DMD) patient in an investigator-initiated trial (IIT). This marks a monumental step in the treatment of DMD, ushering in a new era of gene editing therapy.

Chelsie He, CEO of GenAssist, expressed enthusiasm about this development, stating, "This is the first-in-human trial for DMD gene editing therapy. It marks that DMD treatment has entered the gene editing era." She highlighted the potential of base editors, which are considered the second generation of CRISPR-Cas9 technology, noting their lower off-target risk and immense application potential. He emphasized the devastating impact of DMD on families and the lack of effective treatments available, affirming GenAssist's commitment to leveraging this groundbreaking technology to improve treatments for the DMD population.

GEN6050X Injection Details

GEN6050X is an intravenous cytosine base editing drug designed specifically for DMD patients who are amenable to exon 50 skipping. It employs dual AAV9 vectors and is based on GenAssist's proprietary RNA editing-free Targeted AID-mediated Mutagenesis (TAM) cytosine base editor technology. The drug aims to permanently restore dystrophin expression by editing the mutated DMD gene through a one-shot systemic administration, offering a potential curative solution for DMD.

The investigator-initiated trial is being conducted at Peking Union Medical College Hospital under the identifier NCT06392724. GenAssist is also preparing for the Investigational New Drug (IND) filing of GEN6050X in both the US and China, anticipated for November 2024.

Understanding Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a rare, X-linked recessive genetic disorder caused by mutations in the dystrophin gene. This results in the absence of dystrophin protein, which is crucial for muscle cell membrane stability. DMD primarily affects skeletal and cardiac muscles, leading to progressive muscle degeneration. Symptoms typically appear in early childhood, with affected individuals losing the ability to walk by around age 12. The disease can further lead to severe complications such as heart and respiratory failure, significantly shortening the average life expectancy to around 26 years.

DMD occurs in approximately 1 in 3,500 to 5,000 live male births. According to LEIDEN data, around 80% of DMD patients could potentially benefit from exon skipping therapies, with approximately 4% specifically eligible for exon 50 skipping.

GenAssist Ltd.: A Frontier in Gene Editing

Founded in July 2020, GenAssist Ltd. is a pioneering company in the gene editing drug industry. Based in Suzhou, China, with subsidiaries in Shanghai and Boston, GenAssist holds the global rights to the Targeted AID-mediated Mutagenesis cytosine base editor technology. The company is devoted to developing innovative base editing drugs targeting life-threatening diseases.

GenAssist's progress with GEN6050X marks a pivotal advancement in the treatment of DMD, providing hope for more effective therapies and improved quality of life for those affected by this debilitating condition.

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