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ESGCT 2025: Genethon at the forefront of gene therapy!

From October 7 to 10, 2025, Seville will host the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), the unmissable gathering of the world’s leading gene therapy experts.

Once again this year, Genethon will take center stage, with 19 of its researchers presenting their latest work:

  • 4 oral presentations
  • 19 scientific posters
  • 2 co-chaired sessions by Genethon’s experts

In total, 23 communications highlighting the strength and excellence of Genethon’s research, dedicated to turning scientific breakthroughs into innovative therapies for rare genetic diseases.

“Taking part in the ESGCT Congress is a unique opportunity for our teams to share their most recent results. These scientific and medical advances hold the promise of changing patients’ lives. The selected presentations reflect not only the expertise of our researchers, but also Genethon’s pioneering and recognized leadership in the field of gene therapy.”
Frédéric Revah, Chief Executive Officer of Genethon.

Program highlights

Sessions co-chaired by Genethon

  • Thursday, October 9, 3:30 p.m. – 5:30 p.m. (Room Parallel B)
    Isabelle Richard, PhD, Head of the Progressive Dystrophies Laboratory, will co-chair the session “AAVs/Non-Integrative Vectors II: Mechanism & Biology.”
  • Friday, October 10, 9:00 a.m. – 10:30 a.m. (Room Parallel A)
    Giuseppe Ronzitti, PhD, Head of the Immunology and Liver Disease Laboratory and Director of Scientific Prospective, will co-chair the session “Metabolic Diseases II.”

Oral presentations

Thursday, October 9

  • 8:30 a.m. – 10:30 a.m. (Room Parallel A)

    Giuseppe Ronzitti – “Harnessing the potential of Immunoglobulin G degrading enzymes (Ide) for the treatment of AAV-seropositive patients”
  • 6:00 p.m. – 7:30 p.m. (Room Parallel D)

    Arnaud Valent – “GNT0004, Genethon’s AAV-based gene therapy for Duchenne muscular dystrophy: long-term follow-up of ambulatory boys enrolled in the dose-escalation phase of GNT-016-MDYF”

Friday, October 10

  • 9:00 a.m. – 10:30 a.m. (Parallel Room A)

    Jérémy Do Cao – “Overcoming AAV8 Immunity: First Seropositive Crigler-Najjar Patient Treated with GNT0003 Following Imlifidase Pretreatment (GNT-018-IDES clinical trial)”

  • 9:00 a.m. – 10:30 a.m. (Parallel Room B)

    Lorenzo d’Antiga – “Durability of AAV-based gene therapy in patients with Crigler Najjar syndrome at 4 years of follow-up after dosing: the GNT-012-CRIG Study”

Posters

Wednesday, October 8 – 2:00 p.m. – 3:30 p.m. (Fibes 2)

  • “Preclinical development of GNT0008, a gene therapy product to treat LGMDR1” – Anthony Brureau
  • “11-year survival of a GRMD dog after injection of AAV-microdystrophin gene therapy and post-mortem analysis” – Estelle Creoff
  • “Novel homology-mediated end joining -IDLV precise integration for Therapeutic Genome Editing in Hematopoietic Stem Cells” – Giulia Scalisi
  • “Systemic AAV vector readministration by combination of natural and bioengineered capsids” – Edith Renaud-Gabardos
  • “Mtm1 deficient rats as a new preclinical model for myotubular myopathy gene therapy” – Badih Salman
  • “AAV8-mediated gene replacement corrects the metabolic pathology in a liver-specific mouse model of Glycogen-storage disorder type 1A” – Michael Blatzer
  • “Shuffling of HVR in AAV capsid reveals a context-dependent role for HVR5 in peripheral tissue detargeting while ensuring CNS targeting” – Christian Leborgne
  • “Therapeutic advantage of a Dual AAV-Split Intein MIDI Dystrophin revealed by dynamic muscle function assay in animal and human DMD models” – Maxime Ferrand
  • “Same vectors, different contaminants: tracking HCPs across rAAV serotypes” – Grégory Rouby
  • “The DMDmdx rat: a representative model of Duchenne muscular dystrophy skeletal, cardiac, and respiratory deficiencies” – Estelle Créoff
  • “Accelerating small scale development in gene therapy: Fast and robust full capsids quantification using Mass Photometry Technology from Refeyn (SamuxMP)” – Clotilde Ciesla
  • “Muscle-specific expression reduces early antigen presentation and promotes CD8 T cell tolerance after rAAV gene transfer” – Lindsay Jeanpierre

Thursday, October 9 – 2:00 p.m. – 3:30 p.m. (Fibes 2)

  • “Optidys: a dual-AAV gene therapy strategy for Duchenne muscular dystrophy” – Sonia Albini
  • “Improvement of AAV quality, quantity, and scale-up focusing on the transfection step and dissolved CO2” – Tom Nocerra
  • “Comparison study of intravenous and intracisternal administration in NHP of a rAAV9 gene therapy for acid ceramidase deficiency related disorders” – Michael Hocquemiller
  • “CRISPR activation of utrophin for Duchenne muscular dystrophy therapy” – Paola Galbiati
  • “Metabolic normalization perspective in Duchenne muscular dystrophy” – Elise Lachiver
  • “Quantitative MRI as a Valuable Surrogate Biomarker for Monitoring Disease Progression in DMD: Insights from Genethon’s Multicenter Natural History Study with Standardized Follow-Up” – Arnaud Valent
  • “A second-generation myotropic capsid targeting integrin alpha V beta 6 with enhanced transduction efficacy in skeletal and cardiac muscles” – Ai Vu Hong
For more information, see the full program.