Luis GarciaPrésident at SQY TherapeuticsSpeaker
Profile
Luis Garcia is a pioneer in gene therapies and RNA editing for neuromuscular diseases, combining academic research, industrial partnerships, and clinical applications. For 25 years, he has developed therapeutic approaches based on exon skipping and splicing modulation, using AAV vectors and antisense oligonucleotides. His work, published in Science (2004) and Nature Medicine (2015), has led to major advances, including the creation of SQY Therapeutics and the development of the drug candidate SQY51, currently in Phase 1/2a clinical trials for DMD patients eligible for exon 51 skipping.
He also founded the Inserm Unit UMR1179 (2015-2025) at the UVSQ and co-founded LIA-BAHN (2013–2024), an international laboratory linking the University of Versailles and the Scientific Centre of Monaco, to strengthen scientific cooperation in neuromuscular disabilities. Today, he focuses on building a hospital-university network dedicated to antisense oligonucleotides for rare diseases, in collaboration with APHP R. Poincaré Hospital and SQY Therapeutics.
Agenda Sessions
Interim Pharmacokinetic and Pharmacodynamic Results of the Phase 1/2a AVANCE1 Trial on Tricyclo-DNA-Induced DMD Exon 51 Skipping in Duchenne Muscular Dystrophy
, 16:30View Session