Aurélie Goyenvalle

France

Dr Aurélie Goyenvalle is a scientist focused on gene and antisense therapies for the treatment of neuromuscular disorders. Aurelie received her PhD in Virology at the University of Paris VII in 2006 from her work at Genethon in France, where she developed an exon-skipping gene therapy strategy for Duchenne muscular dystrophy (DMD) using adeno-associated virus vectors (AAV) encoding chimeric U7snRNA constructs carrying antisense sequences. To pursue her interest in gene therapy and splicing modulation approaches for DMD, she joined Prof. Kay Davies’ laboratory at the University of Oxford as a post-doctoral scientist supported by an EMBO fellowship. During her postdoc in Oxford and subsequent position at the Medical Research Council (MRC), Aurélie developed various splicing modulation approaches including the optimization of the U7 snRNA system for neuromuscular and neurodegenerative diseases, and the evaluation of peptide-conjugated antisense oligonucleotides. In 2011, she joined the Institute of Myology in Paris to investigate splice switching approaches using novel antisense oligonucleotides and in 2012, she was awarded a Chair of Excellence program to establish her own group at the University of Versailles to develop novel RNA based technology for the treatment of neuromuscular diseases. In this context, she has notably demonstrated the therapeutic potential of a novel class of AONs made of tricyclo-DNA (tcDNA), which displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration. In 2015, Aurélie has been appointed a permanent research scientist position by the French national institute for health and medical research (INSERM) to pursue this line of research and she is currently head of a team evaluating tcDNA-AON therapeutic potential for various genetic disorders.