Genethon to Present Groundbreaking Gene Therapy Advances in Rome
Genethon is excited to announce the participation of 20 dedicated scientists at the prestigious European Society of Gene & Cell Therapy’s 31st Annual Congress, taking place from October 22 to 25, 2024. This important event offers a platform for Genethon to showcase the forefront of gene therapy research aimed at treating rare and serious diseases.
Sharing Innovative Research
Frederic Revah, Chief Executive Officer of Genethon, expressed enthusiasm about the event. He stated, "Participating in the ESGCT congress is a unique opportunity for our teams to share the latest results of Genethon’s work in the field of gene therapy. Our presentations will highlight both scientific and medical advances that have the potential to significantly improve patients' lives." This underscores Genethon’s commitment to leading the way in the development of transformative therapies.
Key Presentations
At the congress, Genethon will feature several significant oral presentations that represent their cutting-edge research. The schedule includes sessions dedicated to various aspects of gene therapy, focusing on advancements that improve efficacy and safety.
Wednesday, October 23
SESSION 3a: AAV vectors as tools in gene therapy of rare diseases (08:30 to 10:30)
Serge Braun will present data on GNT0004, focusing on the AAV8 vector-delivered microdystrophin gene therapy for Duchenne muscular dystrophy. This presentation will cover the first year's data from the Phase 1/2 clinical trial.
Additionally, SESSION 3d: Metabolic Diseases: Preclinical (08:30 to 10:30) will include insights from Louisa Jauze on liver-directed AAV gene transfer. This research has shown long-term correction of hypoglycemia in a GSDIII mouse model, highlighting the potential impacts of gene therapy.
Thursday, October 24
SESSION 7c: Cardio and Muscular (09:00 to 11:00) will feature Edith Renaud-Gabardos, who will discuss a ten-year efficacy study of gene therapy in a canine model of X-linked myotubular myopathy, demonstrating the persistent benefits of this treatment.
Later in the day, SESSION 9a: in vivo Gene Editing (15:30 to 17:30) will see Maelle Ralu present findings on CRISPR-Cas9 mediated strategies aimed at improving outcomes for patients with Duchenne muscular dystrophy.
Exciting Poster Presentations
In addition to oral presentations, Genethon will showcase 18 posters providing insights into various aspects of gene therapy research. This includes innovations in AAV vectors and their applications in treating genetic conditions.
AAV & Non-integrating vectors research will be highlighted by contributors like Valentina Buffa and Ricardo Rojas Gonzalez, focusing on the development of potency assays and purification processes.
Further, Sonia Albini will unravel the therapeutic efficacy of MIDI dystrophin variants, illustrating the progress in gene therapy for rare muscular diseases.
Contributions in areas such as metabolic diseases show the trajectory towards developing effective treatments for conditions like Farber disease, ensuring that each study presented is positioned to inform and inspire further research and application.
About Genethon
As a pioneer in gene therapy addressing rare diseases, Genethon was founded by AFM-Téléthon. It holds a prominent role in the gene therapy sector, with notable advancements in treatments that continue to change the lives of patients. With a dedicated team of over 200 professionals, the organization is involved in developing a variety of clinical trials for conditions affecting the liver, blood, immune system, muscles, and eyes. Their ongoing commitment aims at producing innovative therapies that radically alter patient care.
Frequently Asked Questions
What is Genethon?
Genethon is a non-profit organization focused on developing gene therapies for rare diseases, established by AFM-Téléthon.
What will Genethon present at the ESGCT congress?
Genethon will showcase oral presentations and posters detailing advancements in gene therapy, particularly for Duchenne muscular dystrophy and metabolic diseases.
Who is leading the presentations?
Leading Genethon's presentations will be key scientists like Frederic Revah, Serge Braun, Louisa Jauze, and others who are experts in their fields.
What types of diseases does Genethon focus on?
Genethon primarily focuses on rare genetic disorders affecting the liver, blood, immune system, muscles, and eyes.
How does Genethon influence gene therapy development?
Genethon impacts gene therapy by driving research and clinical trials dedicated to innovative treatments that improve patient care and outcomes.
