Professor Annemieke Aartsma-Rus is Professor of Translational Genetics in the Department of Human Genetics at Leiden University Medical Center. She played an important role in the development of antisense-mediated exon skipping for Duchenne muscular dystrophy during her doctoral thesis (2000-2004) at the University Medical Center in Leiden (The Netherlands). In December 2007, she became the leader of the “DMD exon skip group”.

Epileptologist and neurologist for children, Stéphane Auvin is Director of the Department of Pediatric Neurology at the Robert Debré University Hospital and also directs the epilepsy program which participates in the development of antiepileptic drugs and clinical trials.

Its clinical and research activities focus on pediatric epilepsy and its treatments. His research team is working on inflammation-epilepsy, the ketogenic diet, and anti-epileptic drugs in the developing brain.
President of the pediatric commission LICE (International League Against Epilepsy), Stéphane AUVIN also acts as associate editor for epilepsy.

After a first part of her career as a DE Masseur-Kinesitherapeute, DO Osteopath and a Master of Science, Mrs Laurence BONNEL was forced to reorient herself following a car accident.
Graduated from the Grande Ecole EDHEC Business School program and then from the Global EDHEC MBA, she is building the second part of her career in the pharmaceutical world.

Commercial leader with more than 15 years of experience in the pharmaceutical field, Laurence Bonnel works in a wide range of therapeutic areas (Oncology, Neurology, Immunology, Endocrinology, CV …) from pre-launch to the maturity stages of the life cycle of the product.

Aurélie Goyenvalle obtained 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 AAV vectors encoding U7snRNA constructs carrying antisense sequences. She then joined Pr. 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), she developed various splicing modulation approaches for neuromuscular and neurodegenerative diseases, including 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. She 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 Goyenvalle was appointed a permanent research scientist at INSERM and in 2020 Director of Research to pursue this line of research. Aurélie Goyenvalle is currently directing the laboratory Biotherapies for neuromuscular diseases at the University of Versailles (UMR 1179) focused on gene and antisense therapies for the treatment of neuromuscular disorders. Dr Goyenvalle received two prestigious awards from the Oligonucleotide Therapeutic Society: the Dr. Alan M. Gewirtz Memorial Scholarship Award in 2011 and the Mary Ann Liebert Publishers, Inc. Young Investigator Award in 2016. She also actively participated in building networks that promote the application of oligonucleotide technology in Europe. As a founding member and management committee member of the European networks “Networking towards clinical application of antisense-mediated exon skipping” (COST Action BM1207) and “Delivery of Antisense RNA therapeutics” (COST Action CA17103), she played a pivotal role in connecting European experts in the field.

Gaëtan Lesca is PU-PH in medical genetics at the CHU de Lyon, specializing in neurogenetics.
He has contributed to the identification of several genes responsible for monogenic epilepsies as well as to the description of genotype-phenotype correlations.
In the diagnostic context, it participates in the transfer of knowledge to develop genetic tests available to epileptology and pediatric neurologists.

He contributed to the constitution of the EPIGENE network and to the definition of the panel of genes for monogenic epilepsies (PAGEM).
He is a reference biologist for the pre-indication “drug-resistant epilepsies with early onset” for the sequencing of the complete genome within the framework of the France Médecine Génomique 2025 plan.

Professor in Pediatric Neurology at Paris Descartes University, doctor in Neuroscience at Pierre and Marie Curie University, Rima Nabbout is a specialist in childhood epilepsy.

Coordinator of the French center for rare epilepsies at the Necker Enfants Malades Hospital, she is also a founding member of the European Reference Network for Rare and Complex Epilepsies (ERN EPICARE).

Originally a research associate at the Pasteur Institute, Frédéric Revah has more than 20 years of experience in the pharmaceutical and biotechnology industries, as well as in academic research.

Head of the Gene Therapy of the Nervous System and Neurochemistry departments at Sanofi from 1992 to 1998, Frédéric Revah then joined the biotech company Cerep as Scientific Director and Managing Director of its oncology subsidiary.

He then joined Sepal Pharma, an innovation company in the field of oncology, before joining the Genethon teams in 2010.