Cellectis to Unveil Breakthrough Gene Therapy Data at European Congress in Rome
October 21, 2024
Cellectis, a clinical-stage biotechnology company, is set to present pre-clinical data at the 31st Annual Congress of the European Society of Gene & Cell Therapy in Rome, Italy, from October 22-25, 2024.
The event will feature four oral presentations and 18 posters that highlight advancements in gene therapies for various diseases.
Among the key presentations, Serge Braun will discuss the Phase 1/2 trial results of an AAV8 vector-delivered microdystrophin gene therapy for Duchenne muscular dystrophy.
Notable oral presentations will cover research on AAV vectors for Duchenne muscular dystrophy, liver-directed AAV gene transfer for glycogen storage disease, and long-term gene therapy efficacy in canine models.
Louisa Jauze will present findings on a gene therapy that corrected metabolic issues in a mouse model of type III glycogenosis, demonstrating long-term benefits.
Taysha Gene Therapies will showcase promising biodistribution data from studies evaluating AAV9 gene therapy delivery methods in non-human primates.
Despite the advancements, scientists continue to face safety and manufacturability challenges in scaling adeno-associated viral gene therapies.
Frederic Revah, CEO of Genethon, emphasized the importance of sharing groundbreaking research that could transform patient care.
Maelle Ralu will highlight CRISPR-Cas9 strategies aimed at enhancing utrophin expression in Duchenne muscular dystrophy models.
Dr. Jean-Philippe Combal will discuss the limitations of current CTX treatment and express optimism for VTX-806 as a potential curative option.
The posters will also cover topics such as AAV vector purification, glycosylation tests, and therapeutic efficacy of dystrophin variants.
Taysha Gene Therapies will present biodistribution data from five studies involving 28 non-human primates at the congress.
Summary based on 11 sources
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Yahoo Finance • Oct 22, 2024
Cellectis to Present Data on TALE-Base Editors and Non-Viral Gene Therapy at the ESGCT 31st Annual Congress