GENETHON France

With 200+ experts, Genethon is pursuing its mission to bring life-changing gene therapy products to patients suffering from rare genetic diseases

[GENETHON France @GenethonFr]

We are delighted to announce the publication in the @nejmunofficial of the long-term benefits from the gene therapy clinical trial for Crigler-Najjar syndrome, developed by @Genethon as part of the international CureCN consortium.
👉 https://t.co/WZ5hzCiFVV
#nejm #crigler

[GENETHON France @GenethonFr]

Don’t miss @GenethonFr exclusive update on our cutting-edge technological platforms and their expanding applications in gene therapy on November 4 by Angela Columbano, PhD, Head Business Development and Partnership, at 1.15 pm and 5.10pm.
@AdTherapiesConf
#GeneTherapy #Biotech

[GENETHON France @GenethonFr]

📌Last day at #ESGCT2026
🎤Barry Byrne will present encouraging Phase 1 results of ATA-200 for #LGMDR5.
🎤 Arnaud Valent will share updated clinical data from the GNT0004 Duchenne muscular dystrophy trial.
Advancing gene therapy from science to patients.
@ESGCT
#GeneTherapy #DMD

[GENETHON France @GenethonFr]

📢Busy day for @GenethonFR at #ESGCT2026!
🎤 Tiziana La Bella will present next-generation enzymes designed to eliminate neutralising antibodies and support repeat dosing in gene therapy.
📌13 posters will also showcase advances in #GeneEditingGene, #AAVengineering

[GENETHON France @GenethonFr]

📌Poster Day at #ESGCT2026
Meet @GenethonFR scientists and discover 8 projects covering:
🧬#AAV engineering, immune evasion, Duchenne muscular dystrophy, clinical research, next-generation vectors.
From GNT0003 to Optidys, join the discussion!
@ESGCT
#GeneTherapy #RareDiseases

[GENETHON France @GenethonFr]

#ESGCT2026 opens today!
💡Isabelle Richard co-chairs the session on AAV & non-integrative vectors.
🎤Giuseppe Ronzitti will address immune responses to AAVs and prospects for redosing.
🎤 Adrian Westhaus will present next-generation AAV vectors to evade pre-existing antibodies.

[GENETHON France @GenethonFr]

J-7 avant #ESGCT2026 à Hambourg !
Les équipes de @GenethonFr présenteront leurs dernières avancées :
🔬 5 communications orales
📌 21 posters scientifiques
🤝 2 sessions coprésidées

👉 https://t.co/gbntEm425z
@ESGCT

#ThérapieGénique #MaladiesRares #Recherche

[GENETHON France @GenethonFr]

One week to go until #ESGCT2026 in Hamburg!
@GenethonFr will share its latest advances with the gene &cell therapy community:
🔬 5 oral presentations
📌 21 posters
🤝 2 co-chaired sessions

👉https://t.co/gbntEm425z

#GeneTherapy #RareDiseases #AAV

[GENETHON France @GenethonFr]

📆First day of the #CGMESA26 ! We look forward to seeing everyone this week. Don’t miss @GenethonFr activities presentation by Imen Mestiri, PhD, Business Development and Licensing Lead October 5th at 9:30 am to 9:45 am, FLW Ballroom F.

#Congress #GeneTherapy #ClinicalTrial #AAV

[GENETHON France @GenethonFr]

@GenethonFr is registered to attend @alliancerm’s #CGMesa26!
Can't wait to dive into discussions, network with industry leaders, and explore the latest advancements in #cellandgenetherapy.
Let the countdown begin!
See you in Phoenix! 🌵🧬

#CGMESA26 #GeneTherapy #ClinicalTrial

[GENETHON France @GenethonFr]

📢 #WMS2026 Sept. 30
🎙️ Oral Session 3 (3:30–4:00 PM)
Mario Amendola: CRISPR-based utrophin upregulation for Duchenne muscular dystrophy
Vincent Laugel: Long-term Phase 1/2 results of GNT0004 gene therapy

📄 3 posters from @GenethonFR (5:15–6:15 PM)

#DMD #GeneTherapy

[GENETHON France @GenethonFr]

📢 Genethon’s Recent Accomplishments Underscore the Progress of Genetic Medicine from Scientific Promise to Clinical Reality
👉To read the full newsletter
https://t.co/gi9skbU5ED
#Duchenne #LGMD #AAV

[GENETHON France @GenethonFr]

📢 @GenethonFr at @Euroocs_conf
On June 24, 🎤 Sonia Albini will co-chair Session 5A and present a novel iPSC-derived organoid model for gene therapy evaluation in Duchenne Muscular Dystrophy.
🗣 Ines Sandid presents her research on fibrosis in DMD organoids.

#GeneTherapy

[GENETHON France @GenethonFr]

We are pleased to announce that @GenethonFr will participate, for the first time at the 3rd Hope for Rare Science Conference in Shanghai !

👉 Full press release : https://t.co/c8MN2kDwlY
#GeneTherapy #RareDiseases #Innovation #Genethon #china #duchenne

Afficher la traduction

[GENETHON France @GenethonFr]

👏Congratulations Djunda Ratenan-Peltier for this well-deserved recognition !
Kudos also to our teams involved and congrats to Valère Desmeure and David ISRAELI for their achievements !

[UF Myology Institute @UFMyology]

And the winner is....🥁(drumroll)... Djunda Ratenan- Peltier!! Congratulations to Djunda for winning the 2026 Communications Award for New Directions 2026. Thank you to everyone who competed and participated with all your likes and shares!

[GENETHON France @GenethonFr]

Proud to see our teams recognized internationally! Congrats to Djunda Peltier, finalist for the #CommunicationAward
@UFMyology
Her project: a new preclinical model + AI tool to better understand and treat calpainopathy.
👉 Like & support her on X:
https://t.co/la3JS4eLr1

[UF Myology Institute @UFMyology]

Announcing the 4 finalist for the New Directions Conference Communication Award (in no particular order)! Vote for your favorite video and the trainee with the most likes wins! @MYOTWlTTER

Finalist #2 of 4: Djunda Peltier

[GENETHON France @GenethonFr]

Fiers de voir nos équipes reconnues à l’international !
Bravo à notre collaboratrice Djunda Peltier, finaliste du #CommunicationAward @UFMyology
👉 Soutenez son projet en likant sur X : https://t.co/la3JS4eLr1

[UF Myology Institute @UFMyology]

Announcing the 4 finalist for the New Directions Conference Communication Award (in no particular order)! Vote for your favorite video and the trainee with the most likes wins! @MYOTWlTTER

Finalist #2 of 4: Djunda Peltier

[GENETHON France @GenethonFr]

📢[COMMUNIQUE DE PRESSE]
@GenethonFr × @AmpersandBio
Un partenariat exclusif pour concevoir des vecteurs AAV à très haute spécificité tissulaire, afin de renforcer la sécurité et l’efficacité des thérapies géniques.
👉https://t.co/LDLPwQDmkm
#GeneTherapy #AAV #Innovation

[GENETHON France @GenethonFr]

📢[PRESS RELEASE]
@GenethonFr × @AmpersandBio
An exclusive partnership to engineer AAV vectors with very high tissue specificity, aiming to enhance the safety and efficacy of gene therapies.
👉 https://t.co/7kIngx1kYQ
#GeneTherapy #AAV #Innovation

[GENETHON France @GenethonFr]

[#ASGCT2026] – Oral presentations
📌Join us for @GenethonFr 's last talks at ASGCT 2026 with:

Gérald Perret, Giuseppe Ronzitti and Paola Galbiati
Advancing clinical gene therapy for rare diseases.

@ASGCTherapy

#GeneTherapy #ClinicalTrials