Alberto Malerba

I was awarded a PhD in Biotechnologies at the Padua University, my research described the role of inflammation in muscle regeneration. Afterwards, I joined the gene therapy laboratory at Royal Holloway, and since then I contributed to the development of novel gene therapy agents and antisense therapeutics for the treatment of muscular dystrophies and other muscle conditions. My initial work was focused on developing antisense reagents for exon skipping of Duchenne muscular dystrophy. I was appointed for 2 years as independent research fellow at the Royal Veterinary College in London, where I developed a scientific program based on new splicing-modulating molecules for the treatment of ischemic diseases in muscle. In 2013, I re-joined the gene therapy laboratory at Royal Holloway as project manager to work on the optimization of gene therapies and antisense therapeutics for a number of muscle diseases. I collaborated on the development of a gene therapy AAV vector for Oculopharyngeal Muscular Dystrophy (OPMD), a rare muscle disease. In December 2023 this vector entered the first-in-human phase I/II clinical trial. I worked within the Unite DMD consortium to complete the preclinical development of an AAV-microdystrophin gene therapy that is now in clinical trial in France and UK. I became a lecturer in gene therapy in 2022 and I am currently leading the Gene Medicine Laboratory for Rare Diseases; our work focuses on developing new genetic treatments for muscular dystrophy and muscle diseases.