Lead, Gene Medicine Laboratory for Rare Diseases · Royal Holloway, University of London · company speakers →
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 has contributed to the development of novel gene therapy agents and antisense therapeutics for the treatment of rare neuromuscular diseases. Between 2011 and 2013 he worked as independent research fellow at the Royal Veterinary College in London, where he developed a scientific program based on new splicing-modulating molecules for the treatment of cardiovascular diseases. In 2013, he re-joined the gene therapy laboratory at Royal Holloway to work as project manager on the optimization of gene therapies for a number of muscular dystrophies. He initially focused on the development of a gene therapy application for Duchenne muscular dystrophy, a rare muscle conditions affecting children. This approach is now in clinical trial in France and UK. Afterwards he developed, in collaboration with a Biopharma company, a gene therapy vector for Oculopharyngeal Muscular Dystrophy, a rare muscle proteinopathy. This vector entered a first-in-human phase I/II clinical trial in 2023. He is now leading the Gene Medicine Laboratory for Rare Diseases at Royal Holloway.
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