July 23, 2025, GenAssist Ltd (GenAssist) announced that the third patient enrolled in the IIT study of GEN6050X at Peking Union Medical College Hospital has successfully completed the 30-day follow-up with no serious adverse events reported. This marks the completion of enrollment for the IIT study.
To date, except the reported SAEs, all three patients in the study have shown no clinically significant symptoms or laboratory abnormalities during follow-up, supporting the early safety profile of GEN6050X. The first patient is scheduled to complete the one-year follow-up in August 2025. Upon completion of the one-year main study, all patients will transition into a 14-year long-term follow-up period.
GenAssist remains committed to transparency and data sharing as it builds on early clinical success and advances next-generation gene therapy programs for Duchenne muscular dystrophy and other rare diseases.
About GEN6050X
GEN6050X is the global first intravenous base editing therapy for DMD, which targets patients amenable to exon 50 skipping. The action mechanism of GEN6050X includes base editor-mediated exon skipping to restore dystrophin expression and ACTG1 overexpression to enhance the cytoskeleton and improve muscle function.
The IIT study is being conducted at Peking Union Medical College Hospital (NCT06392724) since August 2024. All patients in the study received a dose of 5×10¹³ vg/kg. GEN6050X received FDA IND approval on March 6, 2025. GenAssist is preparing to initiate Phase I clinical trial in US.
