The Latest Research of 14 Généthon Scientists to be Featured at the European Society of Gene & Cell Therapy’s 30th Annual Congress, Oct. 24-27, 2023, in Brussels, Belgium

The gene therapy pioneering organization’s research, aimed at curing rare and ultra-rare genetic diseases, is featured in 3 oral and 11 poster presentations.

PARIS, FRANCE (October 24, 2023) – Généthon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today 14 scientists will make oral and poster presentations at the European Society of Gene & Cell Therapy’s 30th Annual Congress October 24 – 27, 2023 in Brussels, Belgium.

“Our scientists will be presenting Généthon’s latest advances in gene therapy research, product development and biomanufacturing,” said Frederic Revah, Genethon CEO. “The breadth of this significant research is testament to the growth in importance of gene therapy in modern medicine. I am proud of the worldwide leadership role of our 200 scientists and professionals who are dedicated to developing new therapies for rare diseases.”

The following are Oral Presentations by Genethon scientists.

Wednesday, October 25

Ai Vu Hong – « An Integrin-targeting AAV developed by a novel computational rational design methodology presents an improved targeting to the skeletal muscle and reduced » presents a rational vector design technology. AAV-based gene therapy requires the injection of a very high dose of vector. This often requires an economic burden and undesirable side effects. By modifying part of the vector capsid, AAV can bind more effectively to muscle cells. This increases the level of gene transfer and targets muscle tissue more effectively, enabling motor function to be restored in animal models. This result demonstrates Généthon’s technological expertise in the design of new-generation AAVs for the treatment of muscle diseases.

Thursday, October 26

Giuseppe Ronzitti – « Innate immunity to AAV vectors: the devil’s in the details » will discuss strategies for improving the safety and long-term persistence of AAV gene transfer in the liver. Although the immune system of this organ appears to be tolerant, several factors can trigger an immune response in the liver. To overcome this obstacle, several strategies for modifying the AAV vector (capsid or genome) are being developed, in conjunction with the administration of immunosuppressive therapy.

Friday, October 27

Maëlle Ralu – « CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy » presents a therapeutic approach for Duchenne Muscular Dystrophy patients, based on the use of another protein called utrophin. The aim is to increase the amount of utrophin in muscles to stabilize it and prevent the dystrophic symptoms. Using the CRISPR-Cas9 tool, it is possible to cut and modify the DNA of cells to change the genetic information that determines the amount of utrophin. In mice carrying Duchenne Muscular Dystrophy, this strategy allowed to increase utrophin levels, and reduce some dystrophic parameters, such as necrosis.