ASGCT 2026: Genethon reinforces Its Scientific Leadership with 21 Presentations, Including 8 Oral Presentations

From May 11 to 15, 2026, Genethon will play a major role at the 29th annual conference of the American Society of Gene and Cell Therapy (ASGCT) in Boston, the world’s leading scientific event dedicated to gene and cell therapy.

With 21 scientific presentations, including 8 oral presentations and 13 posters, Genethon once again demonstrates the depth and diversity of expertise, spanning fundamental research to translational and clinical applications in the field of gene therapy.

View the full program: https://annualmeeting.asgct.org/program

This strong participation reflects Genethon’s central role in developing new gene therapy approaches for rare diseases, to transform scientific innovation into concrete therapeutic solutions for patients.

Highlights

Among the highlights of ASGCT 2026, eight oral presentations will spotlight the most advanced and impactful research programs, underscoring its recognized leadership within the international gene therapy community.

Wednesday, May 13

• 10:15 AM – 10:30 AM – MCEC Room 257AB (Level 2)
  Youssef Krimi Benchekroun – “AAV gene therapy for Glycogen Storage Disease type III with a functional mini-GDE transgene rescues muscle impairment in aged mice and supports clinical translation through dose optimization, NHP and organoid models ” – Presentation of an AAV gene therapy approach that enables muscle cells to produce a functional protein absent in preclinical models of glycogen storage disease type III.
• 10:30 AM – 10:45 AM – MCEC Room 257ABC (Level 2)
  Louisa Jauze – “Liver-directed AAV delivery of an optimized C-terminal FGF23 transgene improves phosphate homeostasis and skeletal mineralization in an X-linked hypophosphatemia mouse model” – Presentation of an AAV-based gene therapy approach targeting the liver to sustainably restore phosphate in the body and improve bone mineralization in a preclinical model of X-linked hypophosphatemic rickets.
• 4:45 PM – 5:00 PM – MCEC Room 210ABC (Level 2)
  Dr. Barry Byrne (Powell Gene Therapy Center, University of Florida) – “Gene therapy for LGMDR5: preliminary safety data from a Phase 1 study of ATA-200” – Presentation of initial results from an AAV gene therapy in the first treated children with limb-girdle muscular dystrophy (LGMDR5), demonstrating safety and good tolerability of the treatment, as well as very encouraging efficacy results.
• Poster Talk II 12:25–12:30 PM – MCEC Exhibit Hall – Presentation Theater Exhibit and Poster Hall (Halls B2–C, Exhibit Level)
  Giulia Scalisi – “Targeted gene addition of lentiviral vector in human hematopoietic stem cells via the homology-mediated end joining pathway” – Presentation of a new strategy for targeted gene integration using lentiviral vectors, enabling the precise and efficient insertion of a therapeutic gene into hematopoietic stem cells, including quiescent ones.

Thursday, May 14

• 9:00 AM – 9:15 AM – MCEC Room 052AB (Exhibit Level)
  Isabelle Richard – “Challenges and solutions in the development of gene therapy for muscular dystrophies” – Explanation of the importance of finely controlling the expression of the therapeutic gene, optimizing the vector, and taking into account the mechanisms specific to each disease to develop safer and more effective treatments for neuromuscular diseases.

Friday, May 15

• 8:00 AM – 9:45 AM – Westin Seaport Commonwealth Ballroom ABC (Concourse Level)
  Gérald Perret – “Long-term follow-up of ambulatory boys treated with GNT0004, Genethon’s AAV-based gene therapy for Duchenne muscular dystrophy: Results from the phase 1/2 part of the GNT-016-MDYF clinical trial” – Presentation of long-term follow-up data for children with Duchenne muscular dystrophy treated with an AAV gene therapy developed by Genethon. The results confirm efficacy at 2 years following treatment at the therapeutic dose, with sustained improvement in muscle function, supported by biological markers and a favorable safety profile.
• 10:15 AM – 10:41 AM – MCEC Room 162AB (Level 1)
  Giuseppe Ronzitti- “Alternate models to overcome neutralization in gene therapy.”
• 4:45 PM – 5:00 PM – MCEC Room 052AB (Exhibit Level) – MCEC Room 052AB (Exhibit Level)
  Paola Galbiati- “CRISPR activation of utrophin as a mutation-independent approach for Duchenne muscular dystrophy therapy” – Presentation of an innovative strategy using CRISPR technology to activate utrophin in Duchenne muscular dystrophy, a protein capable of compensating for the absence of dystrophin. This approach, effective regardless of the mutation causing the disease, demonstrates a significant increase in utrophin levels in muscle and heart models.

13 posters to explore innovation at every stage

Tuesday, May 12 – 5:00 PM – 6:30 PM – MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit Level)

• “Systemic GNT0004 Gene Therapy: Dose-Finding Study in the DMDmdx Rat Model” – [ASGCT 355] Estelle Creoff
• “Integrated Immune Profiling Reveals Transgene-Directed B Cell and Inflammatory Responses in a DMD Patient Experiencing Severe Immune-Mediated Myositis” – [ASGCT 1186] Phillippe Veron
• “Genotoxicity study of CRISPR/Cas9-based genome editing and AAV-transgene targeted integration in HSPC using Long Read-sequencing” – [ASGCT 1368] Alexandra Tachtsidi
• “Next-generation IgG degrading enzymes with improved specific activity, broader specificity across species and reduced antigenicity in humans”- [ASGCT 1830] Rim Harb
• “Intra-CSF administration of an AAV9 vector expressing human acid ceramidase prevents neurological signs in P361R-Farber mice” – [ASGCT 1163] Marion Derome

Wednesday, May 13 – 5:00 PM – 6:30 PM – MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit Level)

• “Mtm1 deficient rats as a new preclinical model for myotubular myopathy gene therapy”- [ASGCT 870] Badih Salman
• “Prime and base editing for the correction of pathogenic valosin-containing protein (VCP) mutations”- [ASGCT 1193] Lydie Debaize
• “Safety and Biodistribution of rAAV9-Mediated ASAH1 Gene Therapy in Nonhuman Primates”- [ASGCT 1220] Jérôme Denard

Thursday, May 14 – 5:00 PM – 6:30 PM – MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit Level)

• “GLP-compliant six-month toxicity and biodistribution study following single intravenous administration of ATA-200 in Sprague-Dawley rats”- [ASGCT 353] Estelle Creoff
• “Internal optimization of the AAV5 capsid for improved transduction”- [ASGCT 1167] Adrian Westhaus
• “Novel AI-designed AAV capsids targeting Integrin alpha V beta 6 shows superior muscle transduction, especially in diaphragm and enables effective low-dose gene therapy in muscular dystrophy models”- [ASGCT 1489] Isabelle Richard
• “Therapeutic challenges in Glycogen Storage Disease type III: how proliferation and inflammation influence rAAV gene transfer stability in a mild fibrotic background”- [ASGCT 1891] Youssef Krimi Benchekroun
• “Impact of pre-existing neutralizing antibodies and IdeS treatment on AAV9 biodistribution in liver, heart and muscle in non-human primates”- [ASGCT 2476] Rim Harb

Through its strong presence at ASGCT 2026, Genethon reaffirms its commitment:

• To advance gene therapy with scientific rigor and excellence,
• To foster collaboration across academic, clinical, and industrial ecosystems,
• To accelerate access to innovative therapeutic innovations for patients with rare diseases