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genethonfr.bsky.social
Genethon France
@genethonfr.bsky.social
Avec plus de 240 scientifiques et professionnels, Généthon a pour objectif de mettre au point des thérapies innovantes qui changent la vie des patients souffrant de maladies génétiques rares.
www.genethon.fr
Une première à l’ESGCT 2025!
Une patiente immunisée contre l’AAV8 a reçu notre thérapie génique GNT0003 grâce à un pré-traitement par imlifidase.
Une avancée majeure pour les patients jusqu’ici exclus des essais cliniques.
urlr.me/gRjHpK
@genethonfr.bsky.social
October 10, 2025 at 8:42 AM
@genethonfr.bsky.social confirms long-term efficacy of its GNT0004 gene therapy for Duchenne muscular dystrophy at @ESGCT & @WorldMuscleSoc
- Improved motor function
- Slowed disease progression
- Sustained reduction in CPK levels
- No serious side effects
urlr.me/kmfg9M
October 9, 2025 at 5:46 PM
@genethonfr.bsky.social confirme l’efficacité à long terme de GNT0004 pour traiter la myopathie de Duchenne
- Fonctions motrices améliorées
- Ralentissement de la progression de la maladie
- Taux de CPK réduits durablement
- Aucun effet secondaire grave
urlr.me/CpYtQe
October 9, 2025 at 5:46 PM
@genethonfr.bsky.social r is registered to attend @alliancerm.bsky.social #CGMesa25! 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! 🌵
#CGMESA #GeneTherapy #ClinicalTrial
October 3, 2025 at 7:55 AM
[ESGCT CONGRES]
🎉 Du 7 au 10 octobre, @genethonfr.bsky.social sera à l'honneur lors du congrès #ESGCT2025 à Séville !
🔬 4 présentations orales, 19 posters – 2 sessions co-présidées.
🌍 Rencontrons-nous!
👉Actualité : urlr.me/aWVrAE
#Genethon #DMD #MaladiesRares #Recherche
October 1, 2025 at 2:05 PM
[ESGCT CONGRESS]
🎉 From Oct 7–10, @genethonfr.bsky.social
takes center stage at #ESGCT2025 in Seville!
🔬 4 oral presentations, 19 contributions – 2 co-chaired sessions
🌍 Let’s connect, share & inspire!
👉Read the full news : urlr.me/G9t7mv
#Genethon #DMD #RareDiseases #Research
October 1, 2025 at 2:02 PM
🌻 International #CriglerNajjar Day
Every day, 10–12h of #phototherapy to survive.
💙 In 2023, first results in @nejm.org confirmed the effectiveness of this innovative #treatment.
👉 Pivotal phase ongoing, now open to children from age 10.
📎 urlr.me/Mj9fNY
June 21, 2025 at 9:37 AM
🌻 💙 Journée internationale #Crigler-Najjar
Chaque jour, #photothérapie 10–12h pour survivre.
💙En 2023, les 1ers résultats dans le
New England Journal of Medicine ont confirmé l’efficacité de ce #traitement innovant.
👉 Phase pivotale en cours, ouverte aux enfants dès 10 ans.
📎https://urlz.fr/nfgw
June 21, 2025 at 9:36 AM
📢 @genethonfr.bsky.social unveiled the 2-year follow-up data of GNT0004 gene therapy trial for #Duchenne Muscular Dystrophy at the @asgct.bsky.social in New Orleans.
#ASGCT2025
🔗urlr.me/DsNnHY
May 17, 2025 at 4:32 PM
BREAKING NEWS
📢@genethonfr.bsky.social a dévoilé aujourd’hui, à #ASGCT2025, les résultats jusqu’à 2 ans de suivi de l’essai clinique de thérapie génique GNT0004 pour la myopathie de #Duchenne.
@asgct.bsky.social
🔗urlr.me/PM6jvR
May 17, 2025 at 4:31 PM
[#ASGCT2025] 📌Today, Saturday May 17, Sonia Albini and Arnaud Valent will be giving oral presentations! Don’t miss their talks on this final day of #ASGCT2025.
#GeneTherapy #RareDiseases #AAV #muscle #ClinicalTrial #DMD
May 17, 2025 at 3:37 PM
[#ASGCT2025] 📌Today, Thursday May 15, don’t miss oral presentations by Isabelle Richard and Maëlle Ralu!
🤝Come and chat with Sonia Albini at her poster [AMA574].
#GeneTherapy #RareDiseases #AAV #muscle #GeneEditing #Organoid
May 15, 2025 at 8:15 AM
[#ASGCT2025] 📌Today, Wednesday May 14 don’t miss Jeanette Zanker’s, oral presentation!

🤝Meet our team at the poster sessions:
Marine Laurent Poster [AMA385],
Arnaud Valent Poster [AMA706]
Franck Gaston Poster [AMA1135]
Christian Leborgne [AMA1138]
#GeneEditing #DMD #DSP
May 14, 2025 at 8:06 AM
[#ASGCT2025]
📌On Tuesday, May 13 Ana Buj-Bello, MD, PhD, from
@genethonfr.bsky.social, will co-chair ”The Advances in Gene Therapy of Neurological Diseases in Small Animal Models” session.
🤝 The opportunity to connect Jeanette Zanker Poster [AMA1431] and Giuseppe Ronzitti Poster [AMA1509].
May 13, 2025 at 1:22 PM
📢 [Scientific Meeting] The 28th Annual #ASGCT2025 Meeting begins Tuesday 13, May 13, in New-Orleans, LA!
@genethonfr.bsky.social
’s research will be featured in five oral presentations and seven posters!
lnkd.in/edAAJpEN
Looking forward to sharing our latest advances in gene therapy!
#GeneTherapy
May 13, 2025 at 11:35 AM
⏰ Good morning fellow #CGMED25 attendees! Day 2 of the conference is underway.
Don’t miss @genethonfr.bsky.social 's activities presentation by Angela Columbano, PhD, Head Business Development and Partnership, April 16th at 3.45pm

#GeneTherapy #RareDiseases #BusinessDevelopment #Genother
April 16, 2025 at 7:35 AM
📢We are pleased to announce, together with I-Stem and @institutdemyologie.bsky.social, the publication of the results using two molecules as potential therapeutic approaches for treating a form of LGMD-R2 in the British Journal of Pharmacology on March 19th.
👉https://urlr.me/bPzaFC
#RareDisease
March 20, 2025 at 10:13 AM
📢Nous annonçons avec I-Stem et l’ @institutdemyologie.bsky.social la publication dans le British Journal of Pharmacology du 19 Mars, de résultats préliminaires sur l’utilisation de deux molécules comme potentielles approches pour traiter une forme de myopathie des ceintures (LGMD-R2).
urlr.me/fSyYQM
March 20, 2025 at 10:10 AM
📢 [congrès SFTCG]
Premier jour du congrès SFTCG-Société Française de thérapie génique et cellulaire à Talence !
stand#7 :Cécilia Lhôte, Responsable de ressources humaines et Claire Bichat Chargée de communication à @genethonfr.bsky.social.
👉 www.genethon.fr
urlr.me/QkRKtv
March 19, 2025 at 2:15 PM
📢 [SFTCG congress]
First day of the congress SFTCG-Société Française de thérapie génique et cellulaire in Talence!
📌booth #7: meet Cécilia Lhôte, Human Resources Manager and Claire Bichat, Communication Officer at @genethonfr.bsky.social .
👉 www.genethon.fr
urlr.me/QkRKtv
March 19, 2025 at 2:13 PM
📢 [SFTCG congress]
From 19 th to 21 th March, nine scientists from @genethonfr.bsky.social will six five oral presentations and display three posters.
🎤🤝Don't miss the oral presentations and posters #11 #13 and #18!
👉https://www.sftcg.fr/event-programme
#research #clinicaltrials #pioneer
March 18, 2025 at 8:40 AM
📢 [congrès SFTCG]
Du 19 au 21 mars, neuf scientifiques de @genethonfr.bsky.social feront six présentations orales et exposeront trois posters.
🎤🤝Ne manquez-pas les présentations orales et les posters #11 #13 et #18 !
👉https://www.sftcg.fr/event-programme
#recherche #essaiclinique #pionier
March 18, 2025 at 8:38 AM
📆Day 1 of the Advanced Therapies Congress, London.
Don’t miss @genethonfr.bsky.social's activities presentations March 18th by Adrian Westhaus, Postdoctoral Researcher at 1:40 pm and Angela Columbano, PhD, Head Business Development and Partnership, at 2:30 pm.
#GeneTherapy #ClinicalTrial #AAV
March 18, 2025 at 8:19 AM
📣We #StandUpForScience
To know more 👉http://standupforscience.fr
March 7, 2025 at 2:27 PM
📆Day 2 of the RE(ACT) Congress in Brussels.
🧬Don’t miss Gerald Perret, Program Director and Clinical Development, @genethon.bsky.social , on March 6th,09h00 to 12h30 pm.
📄#B003 poster is also available about the GNT-014-MDYF natural history study.
#Congress #GeneTherapy #ClinicalTrial
March 6, 2025 at 7:55 AM