Genethon at ESGCT 2026
Oct 27–30, 2026 Hamburg, Germany ~2.5k attendees Website
Genethon brings 25 presentations to ESGCT 2026 in Hamburg. Its work centres on AAV gene therapy for neuromuscular diseases such as Duchenne muscular dystrophy, and on the immune barriers to dosing and redosing. Other strands cover capsid library screening and vector purification for larger-scale production.
| Organization | ESGCT 2026 Attendance |
|---|---|
Genethon Évry, France | |
Non-profit gene therapy research and bioproduction centre created by AFM-Téléthon, developing treatments for rare diseases such as Duchenne muscular dystrophy. | Workshop Tue Oct 27 12:05pm–1:00pm Immune responses and redosing possibilities Vectors and delivery (AAV, lentiviral, LNP)Clinical trials and translation Chair Tue Oct 27 5:00pm–7:30pm SESSION 2a: AAVs/Non integrative vectors I - Basic Vectors and delivery (AAV, lentiviral, LNP) Talk Tue Oct 27 6:15pm–7:30pm Luminal optimization of the VP1/2 N-termini for improved transduction sheds light on AAV5’s trafficking bottlenecks Vectors and delivery (AAV, lentiviral, LNP) Poster Wed Oct 28 1:30pm–3:30pm A platform approach for full capsid enrichment polishing using CIMmultus® PrimaT monolith on different serotypes and transgenes Poster Wed Oct 28 1:30pm–3:30pm Early Results from an Interventional Phase 1 Pediatric Study of ATA‑200 Gene Therapy in LGMDR5 Demonstrate Favorable Safety and Encouraging Pharmacodynamic Activity Poster Wed Oct 28 1:30pm–3:30pm Luminal regions of the AAV VP1/2 N-termini may impact neutralisation by pre-existing antibodies Poster Wed Oct 28 1:30pm–3:30pm Membrane-binding enriched Midi-dystrophin improves muscle function in Duchenne Muscular Dystrophy human organoids and animal models Rare and inherited disease Poster Wed Oct 28 1:30pm–3:30pm Optidys: a dual-AAV gene therapy strategy for Duchenne Muscular Dystrophy Poster Wed Oct 28 1:30pm–3:30pm 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) Poster Wed Oct 28 1:30pm–3:30pm Sustained functional, biomarker, and MRI therapeutic effects following GNT0004 gene therapy in ambulant boys with Duchenne muscular dystrophy: Long-term Phase 1/2 results from the GNT-016-MDYF trial Poster Wed Oct 28 1:30pm–3:30pm Tropism and underlying mechanisms of 2nd generation AAV vectors for neuromuscular diseases Talk Thu Oct 29 9:30am–10:30am Next-generation IgG degrading enzymes with improved specific activity, broader specificity across species and reduced antigenicity in humans Vectors and delivery (AAV, lentiviral, LNP) Poster Thu Oct 29 2:00pm–4:00pm A highly diversified barcoded 10-VR library enables the identification of next-generation capsid variants simultaneously optimized for manufacturing, transduction, and immune evasion Poster Thu Oct 29 2:00pm–4:00pm Blockade of T-independent IgM response reduces induction of anti-AAV IgG Poster Thu Oct 29 2:00pm–4:00pm CRISPR/Cas9 and AAV-mediated gene replacement approach for β-hemoglobinopathies Poster Thu Oct 29 2:00pm–4:00pm GENETH’OFF, a bioinformatic pipeline for CRISPR/Cas9 off-target detection in GUIDE-seq and related integration-based assays Poster Thu Oct 29 2:00pm–4:00pm Gene therapy of spinal muscular atrophy with progressive myoclonic epilepsy by intra-CSF administration of an AAV9-ASAH1 vector Poster Thu Oct 29 2:00pm–4:00pm Impact of downstream purification process and AAV serotype on protein-impurity clearance Poster Thu Oct 29 2:00pm–4:00pm Integrated multi-omics and cross-species analysis identifies candidate biomarkers of gene therapy response in Duchenne muscular dystrophy Poster Thu Oct 29 2:00pm–4:00pm Intracisternal vs intravenous rAAV9 gene therapy for acid ceramidase deficiency related disorders: Safer CNS targeting in NHPs Poster Thu Oct 29 2:00pm–4:00pm Intravenous administration of an engineered myotropic AAVpo1.A1-MTM1 corrects the skeletal muscle pathology of a rat model of myotubular myopathy Poster Thu Oct 29 2:00pm–4:00pm Shuffling of HVRs in AAV capsid reveals a context-dependent role for HVR5/VR-IV in peripheral tissue detargeting while ensuring CNS targeting Poster Thu Oct 29 2:00pm–4:00pm Spatial whole transcriptomics profiling of Duchenne muscular dystrophy biopsies Poster Thu Oct 29 2:00pm–4:00pm Towards clinical translation of an AAV gene therapy for Glycogen Storage Disease type III Poster Thu Oct 29 2:00pm–4:00pm 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 Chair Fri Oct 30 9:00am–10:30am SESSION 11c: Disease models Clinical trials and translation Talk Fri Oct 30 12:45pm–1:00pm Encouraging hepatic safety profile of Genethon’s AAV8 microdystrophin gene therapy (GNT0004): cumulative clinical experience from an ongoing clinical trial in ambulant DMD boys Vectors and delivery (AAV, lentiviral, LNP)Clinical trials and translation |
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