Genethon arrives in force at ASGCT 2025 with 12 communications, including 5 oral presentations
Genethon will participate to the 28th annual meeting of the American Society of Gene and Cell Therapy (ASGCT) from Tuesday, May 13 to Saturday, May 17 in New Orleans. Scientists, researchers, engineers, and medical affairs experts will present their work and advancements through five oral presentations and seven posters, illustrating Genethon’s excellence in gene therapy field.
Tuesday, May 13 – 1:30 PM – 3:15 PM – NOLA Theater C
Ana Buj-Bello, MD, PhD, Director of the mixed research unit and Head of Neuromuscular Diseases and Gene Therapy, will co-chair the session: “Overcoming Challenges in Efficient Delivery of Gene Therapy to Muscle and Bone”.
Oral presentations
Wednesday, May 14
8:00 AM – 9:45 AM – NOLA Theater A
Jeanette Zanker – « Understanding subcellular trafficking of natural and engineered AAV capsids by novel imaging techniques in skeletal muscle cells » – Presentation of innovative imaging techniques to decipher the intracellular subcellular of AAV capsids in muscle: a promising approach to optimize gene therapies for muscular dystrophies.des dystrophies musculaires.
Thursday, May 15
8:00 AM – 9:45 AM – NOLA Theater B
Isabelle Richard – « Bypassing issues relative to capsid and transgene expression in AAV-mediated transfer for Muscular Dystrophies » – Présentation de stratégies possibles afin de réduire la toxicité observée lors d’essais cliniques, en améliorant les capsides AAV ou en permettant une régulation du transgène.
1:30 PM – 3:15 PM – Room 288-290
Maëlle Ralu – « CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy » – Use of a CRISPR-Cas9 strategy in mice suffered from Duchenne muscular dystrophy, to overexpress utrophin, a protein that can compensate for the absence of dystrophin, thereby improving the phenotype.
Saturday, May 17
8:00 AM – 9:45 AM – NOLA Theater
Sonia Albini- « Optidys: A Dual-AAV Gene Therapy Strategy For Duchenne Muscular Dystrophy» – Presentation of preclinical data on a new AAV capsid (LICA) combining a dual AAV system and a long-sequence quasi-dystrophin in the context of gene therapy for treating Duchenne muscular dystrophy.
10:15 AM – 12:00 PM – Room 288-290
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» – Presentation of 2-year follow-up data of GNT0004 gene therapy trial for Duchenne Muscular Dystrophy.
Posters
Tuesday, May 13 – 6:00 PM – 7:30 PM – Poster Hall Hall 12
- “Understanding DNA host cell factors as potential restriction factors to efficient AAV gene transfer in skeletal muscle cells” – [AMA 1431] Jeanette Zanker
- “Long-term correction of metabolic impairment in a mouse model of Glycogen Storage Disease type III after rAAV gene transfer reveals disease-specific host-vector interactions” – [AMA 1509] Giuseppe Ronzitti
Wednesday, May 14 – 5:30 PM – 7:00 PM – Poster Hall Hall 12
- “Optimizing Gene Editing for Erythroid-Specific Therapeutic Protein Expression Using HSPCs”- [AMA 385] Marine Laurent
- “Comprehensive analysis of longitudinal SV95C measurements, an e-digital mobility assessment in a real-life DMD population in the GNT-014-MDYF natural history study” – [AMA706] Arnaud Valent
- “Development of a full capsid enrichment polishing step in AAV8 purification process with CIMmultus® PrimaT® monolithic column”- [AMA 1135] Franck Gaston
- “Rational shuffling: a novel capsid engineering method to unlock the translational potential of wild-type Adeno-associated virus”- [AMA 1138] Christian Leborgne
Thursday, May 15 – 5:30 PM – 7:00 PM – Poster Hall Hall 12
- “Therapeutic screening of Dual AAV-Split Intein MIDI dystrophins by analysis of mechanical properties in vivo and in human muscle organoids”- [AMA 574] Sonia Albini
