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Genethon at ESGCT 2026: 20 experts, 25 major breakthroughs, one shared goal: to accelerate the gene therapy revolution.

At the 33rd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) in Hamburg, Genethon will present 25 scientific presentations covering the entire gene therapy value chain, from basic research to clinical and industrial development.

The selected presentations will highlight significant advances in strategic areas such as AAV vector engineering, genome editing, immunology, biomarkers, innovations in bioproduction, and the results of ongoing clinical trials.

These advances reflect Genethon’s ambition: to advance gene therapy at every stage of its development to provide new solutions for patients who currently have no treatment options. 

This recognition is also reflected in the appointment of two Genethon experts as co-chairs of major scientific sessions: Isabelle Richard, PhD, DRCE CNRS, Head of the Muscular Dystrophies Laboratory, will co-chair a session titled “AAVs/Non-Integrative Vectors I – Basic,” and Mario Amendola, PhD, Head of the Gene Editing Laboratory, will co-chair a session titled “Disease Models.”

“With 25 scientific presentations, including five selected oral presentations and the co-chairing of two scientific sessions by Isabelle Richard and Mario Amendola, Genethon reaffirms its role as an international leader in gene therapy. These results reflect the excellence of our teams and their commitment to pushing the boundaries of innovation to develop increasingly effective and accessible treatments for patients with rare diseases. Every advance presented at ESGCT helps accelerate the transformation of scientific discoveries into concrete therapeutic solutions.” Frédéric Revah, CEO of Genethon.

Program Highlights

Sessions Co-chaired by Genethon

  • Tuesday, October 27, 5:00 pm – 7:30 pm (Parallel – Hall 1, Levels 2, 3 & 4)
    Isabelle Richard, PhD, Head of the Progressive Dystrophies Laboratory, will co-chair the session “AAVs/Non-Integrative Vectors I: Basic.”
  • Friday, October 30, 9:00 am – 10:30 am (Parallel Session – Hall 3, Level 0)
    Mario Amendola, PhD, Head of the Gene Editing Laboratory, will co-chair the “Disease Models” session.

Oral Presentations

Tuesday, October 27

  • 11:15 am –1:00 pm (Hall 3, Level 0) Giuseppe Ronzitti – “Immune Responses and Redosing Possibilities.”
    Focus on a key challenge in in vivo gene therapy: the immune response to AAV vectors. A presentation of innovative approaches aimed at enabling the safe readministration of treatments and enhancing their long-term efficacy.
  • 5:00 pm – 7:30 pm (Hall 1, Levels 2, 3 & 4) Adrian Westhaus – “Luminal optimization of the VP1/2 N-termini for improved transduction sheds light on AAV5’s trafficking bottlenecks.”
    This presentation will explore new AAV vectors capable of largely evading pre-existing antibodies and improving transduction of multiple targets, with a focus on changes in intracellular trafficking that account for their increased efficacy.

Thursday, October 29

  • 8:30 am – 10:30 am (Hall 1, Levels 2, 3 & 4) Tiziana La Bella – “Next-generation IgG-degrading enzymes with improved specific activity, broader specificity across species, and reduced antigenicity in humans.” 
Presentation of a new generation of enzymes capable of more effectively eliminating neutralizing antibodies, a promising advancement for facilitating the re-administration of gene therapies.

Friday, October 30

  • 9:00 am – 10:30 am (Hall 1, Levels 2, 3, & 4) Barry Byrne – “Early Results from an Interventional Phase 1 Pediatric Study of ATA-200 Gene Therapy in LGMDR5 Demonstrate Favorable Safety and Encouraging Pharmacodynamic Activity.” Presentation of the first clinical results for ATA-200, the gene therapy developed for SGCG-related limb-girdle muscular dystrophy (LGMD-R5). After one year of follow-up, the pediatric Phase I study demonstrates a favorable safety profile and encouraging signs of biological activity in the first children treated.
  • 11:00 am – 1:00 pm (Hall 1, Levels 2, 3 & 4)  Arnaud Valent “Encouraging hepatic safety profile of Genethon’s AAV8 microdystrophin gene therapy (GNT0004): cumulative clinical experience from an ongoing clinical trial in ambulatory DMD boys.” Presentation of clinical data from patients with Duchenne muscular dystrophy treated with GNT0004, particularly from the pivotal phase of the GNT-016-MDYF study, demonstrating an encouraging safety profile under transient immunosuppression.

Posters

Wednesday, October 28 – 1:30 pm – 3:30 pm

  • « Overcoming AAV8 Immunity: One-year follow-up of the first seropositive Crigler–Najjar syndrome patient treated with GNT0003 following imlifidase pretreatment (GNT-018-IDES clinical trial) » – Ji Gao Desliens
  • « Luminal regions of the AAV VP1/2 N-termini may impact neutralisation by pre-existing antibodies » – Adrian Westhaus
  • « A platform approach for full capsid enrichment polishing using CIMmultus® PrimaT monolith on different serotypes and transgenes » – Franck Gaston
  • « Membrane-binding enriched Midi-dystrophin improves muscle function in Duchenne Muscular Dystrophy human organoids and animal models» – Sonia Albini
  • « Optidys: a dual-AAV gene therapy strategy for Duchenne muscular dystrophy » – Sonia Albini
  • « Tropism and underlying mechanisms of 2nd generation AAV vectors for neuromuscular diseases » – Nissaï Beaude
  • « Early Results from an Interventional Phase 1 Pediatric Study of ATA‑200 Gene Therapy in LGMDR5 Demonstrate Favorable Safety and Encouraging Pharmacodynamic Activity » – Isabelle Richard

Thursday, October 29 – 2:00–4:00 pm

  • « CRISPR/Cas9 and AAV-mediated gene replacement approach for β-hemoglobinopathies » – Alexia Spinu
  • « Intravenous administration of an engineered myotropic AAVpo1.A1-MTM1 corrects the skeletal muscle pathology of a rat model of myotubular myopathy» – Louise Mangin
  • « Integrated multi-omics and cross-species analysis identifies candidate biomarkers of gene therapy response in Duchenne muscular dystrophy » – Aleksandr Balatskii
  • « Spatial whole transcriptomics profiling of Duchenne muscular dystrophy biopsies » – Amanda Finan-Marchi
  • « A highly diversified barcoded 10-VR library enables the identification of next-generation capsid variants simultaneously optimized for manufacturing, transduction, and immune evasion » – Ai Vu Hong
  • « Blockade of T-independent IgM response reduces induction of anti-AAV IgG » – Coralie Pecquet
  • « Shuffling of HVR in AAV capsid reveals a context-dependent role for HVR5 in peripheral tissue detargeting while ensuring CNS targeting » – Tiziana La Bella
  • « Two-year biological and clinical effects following ATA-100, an AAV-FKRP, gene transfer in LGMDR9: dose-escalation results from a phase 1/2 study » – Isabelle Richard 
  • « GENETH’OFF, a bioinformatic pipeline for CRISPR/Cas9 off-target detection in GUIDE-seq and related integration-based assays » – Guillaume Corre
  • « Intracisternal vs intravenous rAAV9 gene therapy for acid ceramidase deficiency related disorders: Safer CNS targeting in NHPs » – Michael Hocquemiller
  • « Towards clinical translation of an AAV gene therapy for Glycogen Storage Disease type III » – Jérémy Rouillon
  • « Gene therapy of spinal muscular atrophy with progressive myoclonic epilepsy by intra-CSF administration of an AAV9-ASAH1 vector » – Ana Buj-Bello
  • « Impact of downstream purification process and AAV serotype on protein-impurity clearance » – Grégory Rouby