Cellectis to Unveil Breakthrough Gene Therapy Data at European Congress in Rome

October 21, 2024
Cellectis to Unveil Breakthrough Gene Therapy Data at European Congress in Rome
  • 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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