Dr Malerba was awarded a PhD in Biotechnologies, describing novel approaches to improve muscle regeneration. He joined the gene therapy laboratory at Royal Holloway, University of London, in 2007 and since then he contributed to the development of novel gene therapy agents and antisense therapeutics for the treatment of rare diseases. His work was instrumental in optimizing the dosing regimens used for the morpholino exon skipping phase II/III clinical trial for Duchenne muscular dystrophy. Afterwards, he worked for 2 years as independent research fellow at the Royal Veterinary College in London, where he conceived and developed a scientific programme based on new antisense molecules for the treatment of cardiovascular diseases. In 2013, he re-joined as project manager the gene therapy laboratory at Royal Holloway where he has been working on the optimization of a gene therapy medicinal product for Duchenne muscular dystrophy. He is also supervising the work on new drugs and gene therapy approaches for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) a rare muscle proteinopathy. His work was crucial in the development of a gene therapy AAV vector in collaboration with Benitec Biopharma that has been recently granted orphan drug status by EMA and FDA and is expected to enter a first-in-human phase I/II clinical trial in 2019.