Hangzhou-based clinical stage gene therapy firm Exegenesis Bio has announced receiving approval from the US FDA to conduct a Phase I clinical study for its EXG102-031. The study will assess the safety, tolerability, and vision improvement potential of this once-administered gene therapy in patients with wet age-related macular degeneration (wAMD).
Disease Background and Unmet Needs
Age-related macular degeneration (AMD) is a progressive eye disease caused by damage to the central macula of the retina, leading to blurred vision and blindness. In China alone, over 50 million people suffer from AMD, with approximately 5 million having the neovascular form known as wet AMD (wAMD). Globally, wAMD accounts for about 90% of AMD-related blindness, highlighting the significant unmet need for effective treatments.
Exegenesis Bio’s Technology and Pipeline
Exegenesis Bio is built around artificial intelligence (AI)-based platforms and capabilities in virus capsid, promoter, and unique protein engineering design. The firm is also developing EXG001-307, an AAV gene therapy for type I spinal muscular atrophy (SMA), which is currently in clinical stages. The approval for EXG102-031 marks a significant step forward in the company’s efforts to address vision-threatening diseases through innovative gene therapies.-Fineline Info & Tech
