BSGCT Webinar – Gene therapy for neuromuscular conditions

We are thrilled to have Dr. Alberto Malerba as our next guest speaker, presenting his work on gene therapies for neuromuscular conditions.

Dr Malerba holds a PhD in Biotechnologies, awarded at the University of Padua. He joined the gene therapy laboratory at Royal Holloway, University of London, in 2007 to work as postdoctoral scientist In the laboratory directed by Prof George Dickson. Since then he has worked on the development of novel gene therapy agents and antisense therapeutics for the treatment of rare muscle diseases. He initially focused on accelerating the clinical translation of exon skipping for Duchenne muscular dystrophy (DMD). Afterwards, as Research officer and project manager of Dickson’s lab, he collaborated on the development of second-generation gene therapy product for DMD, currently in clinical trial. He has also worked on new drugs and gene therapy approaches for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) a rare muscle proteinopathy. In this context he has developed a gene therapy AAV vector in collaboration with Benitec Biopharma. The vector has recently entered a first-in-human phase I/II clinical trial in US. He is Lecturer since 2022 and he is currently leading the Gene medicine laboratory for rare diseases.