Genethon’s research will be featured in 4 oral presentations and 10 posters at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), May 16-20, 2023, at the Los Angeles Convention Center in Los Angeles, CA.
“We are proud to report some of our most recent progress, covering the advancement of novel gene therapy candidates for multiple rare genetic diseases, and the design of novel vectors and tools,” said Frederic Revah, Genethon CEO. “The objective of Genethon’ scientists is to pursue research of excellence allowing more and more patients with rare genetic diseases to benefit from the treatments they need.”
Genethon’s Ana Buj Bello, M.D., Ph.D., MBA, leader of the Neuromuscular Diseases and Gene Therapy Team, is co-chair of the oral abstract session on AAV Vectors – Clinical/Nonhuman primate studies, Wednesday, May 17, 3:45 pm – 5:30 pm US PDT (Rm 411).
Dr. Buj Bello also is presenting a poster titled, AAV-mediated gene therapy corrects the severe phenotype of acid ceramidase deficient mice (Board No. 1598), Friday, May 19, 12 pm – 2 pm US PDT.
Genethon has four scientists participating in the following oral poster presentation sessions (all times are US PDT):
Wednesday, May 17
AAV Vectors – Preclinical and Proof-of-Concept In Vivo Studies I (3:45 pm – 5:30 pm) Rm 408 AB
• More Than Nine Year Survival of a GRMD Dog after Injection of AAV-Microdystrophin Gene Therapy (4:15 pm – 4:30 pm) – Caroline Le Guiner
Thursday, May 18
Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases I (1:30 pm to 3:15 pm) Rm 403 AB
• Single AAV vector gene therapy with a mini-GDE transgene corrects muscle impairment in mouse, rat and human cellular models of glycogen storage disease Type III (3 pm – 3:15 pm) – Antoine Gardin
Novel Genetic Approaches for Muscle Diseases (3:35 pm – 5:30 pm) Rm 408 AB
• Development of MYOrganoids for therapeutic evaluation of gene therapy product in Duchenne Muscular Dystrophy (4:30 pm – 4:45 pm) – Laura Palmieri
Saturday, May 20
AAV Development for Eye, Muscle, Kidney and CNS (8 am – 9:45 am) Concourse Hall 150 & 151)
• An integrin-targeting AAV developed by a novel computational rational design methodology presents an improved targeting to the skeletal muscle and reduced tropism to the liver (9:25 am to 9:42 am) – Ai Vu Hong
In addition, Dr. Hong, a member of Genethon’s Progressive Muscular Dystrophies Team, received two ASGCT awards: one for Excellence in Research, which recognizes the top 10 – 15 posters submitted by students and fellows; and the other a Meritorious Abstract Travel Award given to associate members based on their high abstract scores.
The following are poster presentations by Genethon scientists (all times are US PDT):
Wednesday, May 17, 12 pm – 2 pm
Extreme Inter-Individual Variability of Subretinal AAV-Induced Immune Response Even in a Highly Standardized Context [Board No. 721] – Duohao Ren and Sylvain Fisson
Assessment of Therapeutical Potential and Advantage of a Dual AAV Approach for Duchenne Muscular Dystrophy [Board No. 663] – Sonia Albini
In Vivo Gene Therapy for Lysosomal Acid Lipase Deficiency [Board No. 643] – Marine Laurent
Characterization and Quantification of CRISPR/Cas9-Induced Genomic Alterations and AAV Targeted Integration in Human HSPC [Board No. 527] Alexandra Tachtsidi
Thursday, May 18, 12 pm – 2 pm
Evaluation of Gene Transfer Efficiency in a Mild Model of Dystrophic Muscle Disorder Performed by Machine Learning and Linear Discriminant Analysis [Board No. 1214] – Anthony Brureau
Cardiomyocyte Death after AAV Gene Transfer Can Be Related to Proteotoxicity or to Transgene Abnormal Function or Localization [Board No. 884] – Isabelle Richard
Predictive Potential of Single Deleterious Variations to Infer on AAV2 and AAV2-13 Capsids Viability [Board No. 874] – Tiziana La Bella
Friday, May 19, 12 pm – 2 pm
Autophagy Induction Improves Dual AAV Vector Efficacy in a Mouse Model of Glycogen Storage Disease Type III [Board No. 1396] – Louisa Jauze
Preliminary Results from a Prospective, Multicentric, Follow Up Standardized Cohort to Assess Natural History of Duchenne Muscular Dystrophy [Board No. 1603] – Fei Cao
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