📢 [#ESGCT2024] 🎤Today from 08:30 am - 10:30 am CEST time. Serge Braun presents “GNT0004, Genethon’s AAV8 vector-delivered microdystrophin Gene Therapy of Duchenne Muscular Dystrophy, first data from phase 1/2 part of the GNT-016-MDYF all-in-one clinical trial in ambulant boys.”

📢 [ESGCT2024] 👏Thank you Serge Braun, Director of the neuromuscular strategy GENETHON France for this presentation of one-year data from the Phase ½ clinical trial on GNT0004. #genethon #congress #ESGCT2024 #presentation #DMD #ClinicalTrial

📢 [#ESGCT2024] 👏Thank you Louisa Jauze, Research fellow @GenethonFR for presenting the development of a gene therapy that corrected the hepatic symptoms of type III glycogenosis up to 9 months after injection in an animal model of the disease. #congress #presentation #liver

📢 [#ESGCT2024] 📄Today from 01:30 pm - 03:00 pm CEST time - Don’t miss Ricardo Rojas-Gonzales from @GenethonFR [Board No.0012]: Development of a full capsid enrichment polishing step in AAV8 purification process with CIMmultus® PrimaT monolithic column. #AAV #DSP

📢 [#ESGCT2024] 📄Today from 01:30 pm - 03:00 pm CEST time – Don't miss Louise Mangin from GENETHON France [Board No.0118]: The engineered AAVpo1.A1 vector transduces efficiently murine and human skeletal muscle fibers with liver detargeting in a model of X-linked myotubular myopathy

📢 [#ESGCT2024] 📄Today from 01:30 pm - 03:00 pm CEST time -Come and ask questions to Sonia Albini from GENETHON France about her poster: Therapeutic efficacy of MIDI dystrophin variants via split intein dual AAV approach, [Board No.0124]

📢 [#ESGCT2024] 📄Today from 01:30 pm - 03:00 pm CEST time - Don’t miss Sophie Olivier. She will present a poster with @GenethonFR about: Natural history of limb girdle muscular dystrophy R9: two-year follow-up of a European cohort, [Board No.0264].

📢 [#ESGCT2024] 📄Today 01:30 pm - 03:00 pm CEST time – Don’t miss Emmanuel Thevenot , @GenethonFR [Board No.0088].

📢 [#ESGCT2024] 📄Today from 01:30 pm - 03:00 pm CEST time - Don’t miss Marion Derome from GENETHON France , with her poster: CSF-directed administration of an AAV9-ASAH1 vector to prevent acid ceramidase deficiency in newborn P361R-Farber mice, [Board No.0300]

📢 [#ESGCT2024] 📄Today from 01:30 pm - 03:00 pm CEST time -Come and ask questions to Prof. Dr. Odile Boespflug Tanguy, from I-motion Institute with her poster: Death after intravenous rAAV9 gene therapy for acid ceramidase deficiency in a SMA-PME patient, [Board No.0432]

📢 [#ESGCT2024] 🫵SAVE THE DATE - Today from 8:30 am - 10:30 pm CEST time Don’t miss the oral presentation about Cardio and Muscular session: “Ten-year efficacy of gene therapy in a canine model of X-linked myotubular myopathy “, by Edith Renaud, Research fellow GENETHON France.

📢 [#ESGCT2024] 👏Thank you Edith Renaud Gabardos, Research fellow @GenethonFR for presenting your work on”Ten-year efficacy of gene therapy in a canine model of X-linked myotubular myopathy”. #presentation #MTM

📢 [#ESGCT2024] 📄Today from 02:00 pm - 03:30 pm CEST time - Don’t miss Jérôme Denard from GENETHON France with his poster: Long-term correction of acid ceramidase deficiency by intravenous AAV-mediated gene therapy in presymptomatic P361R-Farber mice, [Board No.0893].

📢 [#ESGCT2024] 📄Today from 03:30 pm - 05:30 pm CEST time - Don’t miss the oral presentation about in vivo Gene Editing in Preclinical Models: “CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy “ by Maëlle Ralu, PhD Student GENETHON France.

📢 [#ESGCT2024] 📄Today from 06:00 pm - 07:30 pm CEST time - Come and meet Amandine François from GENETHON France, with her poster: A novel AAV gene therapy strategy targeting the liver to treat bone and dental defects in X-linked hypophosphatemia,[Board No.1010]

📢 [#ESGCT2024] 📄Today from 06:00 pm - 07:30 pm CEST time - Come and meet Alexandra Tachtsidi from GENETHON France with her poster: CRISPR/Cas9-induced genomic alterations and AAV targeted integration in HSPC through the lens of Long Read-sequencing, [Board No.0602]. #GeneEditing

📢 [#ESGCT2024] 📄Today 06:00 pm - 07:30 pm CEST time - Martina Marinello GENETHON France presents [Board No.0890]: Dose-finding and toxicological studies of intravenous administration of an AAV9-ASAH1 vector for Farber disease and spinal muscular atrophy with progressive epilepsy.

📢 [#ESGCT2024] 📄Today 06:00 pm - 07:30 pm CEST time - Come and ask your questions to Anne Galy with her poster: Reduction of muscle fibrosis by FAP-CAR T cells enhances rAAV gene transfer efficiency in a murine model of Duchenne muscular dystrophy. [Board No.0734] #DMD

📢 [#ESGCT2024] 👏Thank you Maëlle Ralu PhD GENETHON France for presenting your work about using of a CRISPR-Cas 9 strategy, in the context of DMD in mice, to over-express utrophin in cells, a protein that can compensate for the absence of dystrophin, and thus improve the phenotype.

📆Day 1 of the @AdTherapiesCongress , Philadelphia. Don’t miss GENETHON France activities presentation by Angela Columbano, PhD, Head Business Development and Partnership, November 12th at 3:30 pm, Room 120C. #Congress #GeneTherapy #ClinicalTrial #AAV #DMD