Approximately 1 in 2500 neonates develop cholestatic jaundice, often due to rare genetic disorders with no cure and severe morbidity. This talk will explore how innovative lentiviral gene therapies could transform treatment, using ARC syndrome as a case study. We will highlight key breakthroughs in optimizing gene delivery and ensuring vector safety— crucial steps toward life-changing therapies.

Dr Claudiu Cozmescu is a postdoctoral researcher in the Genetics and Genomic Medicine Research Teaching Department at the UCL Institute of Child Health, focused on advancing in vivo lentiviral gene therapy for inherited liver and multi-organ disorders. He leads cutting-edge projects, including “Multi-organ lentiviral gene therapy for ARC Syndrome,” funded by LifeArc, and has secured funding from Action Medical Research and the National Institute for Health and Care Research as PI and Co-PI to develop novel treatments for related genetic conditions. With a strong background in gene therapy and genetic technologies, Dr Cozmescu’s work bridges innovative research with real-world clinical applications. He also helps shape the next generation of scientists as Co-Lead of two MSc core modules at the UCL Institute of Child Health: “Molecular Aspects of Cell and Gene Therapy” and “Clinical Applications of Cell and Gene Therapy.”

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