Currently, thirteen of Genethon's products are in clinical trials, with an additional seven expected to enter trials in the next five years.

The integration of Asimov's clonal HEK293 cell line with optimized two-plasmid systems can yield unconcentrated titers of up to 1E12 viral genomes per milliliter across various serotypes.

Genethon, a pioneering French laboratory established by AFM-Telethon, will present groundbreaking research at the European Society of Gene & Cell Therapy's 31st Annual Congress in Rome from October 22 to 25, 2024.

The event will showcase four oral presentations and 18 posters highlighting advancements in gene therapies for various diseases.

Mark Stockdale will discuss the role of synthetic biology and artificial intelligence in enhancing the safety, potency, and manufacturability of gene therapies.

Maelle Ralu will present CRISPR-Cas9 strategies that enhance utrophin expression in Duchenne muscular dystrophy models, potentially improving patient outcomes.

In addition to the oral presentations, 18 posters will be presented by Genethon collaborators on topics such as AAV vector purification and therapeutic efficacy of dystrophin variants.

Genethon is recognized for its leadership in developing gene therapies for rare diseases, employing over 200 professionals dedicated to innovative treatments.

Frederic Revah, CEO of Genethon, emphasized the significance of sharing their latest research results, which have the potential to transform patient care.

Serge Braun will kick off the congress with a presentation on the Phase 1/2 clinical trial results of an AAV8 vector-delivered microdystrophin gene therapy for Duchenne muscular dystrophy.

Louisa Jauze will share findings on a gene therapy that corrected metabolic issues in a mouse model of type III glycogenosis, demonstrating long-term benefits.

A live webinar on miniaturized plasmid technology, hosted by VectorBuilder, will take place on November 14, 2024, providing insights into advancements in gene delivery.