GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in genome medicines, announced that it has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for GEN6050X, a first-in-class base editing drug for Duchenne muscular dystrophy (DMD). GenAssist is planning to conduct clinical study globally for GEN6050X.
“US FDA clearance of our first IND is a significant milestone for our company. It validates the ability for our Targeted AID-mediated Mutagenesis (TAM) cytosine base editor technology to target diseases previously considered untreatable—broadening the full-body application of gene editing technology. As the second generation of CRISPR-Cas9, base editors offer immense potential with significantly lower off-target risks.” said Dr. Chunyan He, CEO of GenAssist, “GenAssist is the first company to apply gene editing drug for Duchenne muscular dystrophy indication. By permanently repairing the mutated DMD gene, base editing may provide long-term benefit for DMD patients. We are excited about the potential of this program to bring new treatment for patients and demonstrate gene editing as a new therapeutic approach for DMD. In addition, GenAssist is advancing other Duchenne muscular dystrophy exon-skipping programmes, which cover more than 30 per cent Duchenne muscular dystrophy population.”
GEN6050X injection is an intravenous cytosine base editing drug designed for Duchenne muscular dystrophy 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 may permanently restore the expression of dystrophin through editing the mutated DMD gene. It provides an alternative solution for Duchenne muscular dystrophy patients. The IIT study is being conducted at Peking Union Medical College Hospital (NCT06392724) since August 2024. Until now, two patients have been dosed. The first 10-year-old patient has finished six-month follow-up.
Safety update of GEN6050X for the first two participants dosed: The drug was safe and tolerant with only transient and manageable SAEs; No SUSARs observed; No hospitalizations reported; All treatment-related AEs resolved with no sequelae; No AEs of hepatic transaminitis observed, including no elevated gamma-glutamyl transferase levels.
Duchenne muscular dystrophy 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. Duchenne muscular dystrophy primarily affects skeletal and heart muscles, with progressive muscle wasting symptoms leading to loss of ambulation around age 12. Notably, patients reach a peak NSAA score of 26 at around 6.3 years old, after which the score declines by about 3 points per year. As the disease advances, further complications, including heart and respiratory failure, occur, ultimately reducing the average life expectancy to about 26 years.
Duchenne muscular dystrophy affects approximately 1 in 3,500 to 5,000 live male births. According to LEIDEN data, about 80 per cent of Duchenne muscular dystrophy patients could potentially benefit from exon skipping, with 4 per cent specifically eligible for exon 50 skipping.