Genethon’s R&D to be Featured in Multiple Presentations at American Society of Gene & Cell Therapy Annual Meeting May 16-20, 2022, in Los Angeles, CA

10 May 2023
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Gene TherapyClinical Study
Genethon is a leader in developing gene therapies for rare and ultra-rare diseases PARIS, FRANCE (May 10, 2023) – Genethon, a unique non-profit gene therapy R&D, announced today its 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. The international convention attracts thousands of scientists, physicians, patient advocates and government officials. “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. 15918), 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 About Genethon A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a unique non-profit organization created by a patient association, the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has obtained marketing for spinal muscular atrophy. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases. 13 products resulting from Genethon’s research are in clinical trials for eye, liver, blood, immune system and muscle diseases. A further6 products are in the preparation phase for clinical trials over the next five years. Find out more: genethon.com Contact: Charles Craig Opus Biotech Communications 404-245-0591 charles.s.craig@gmail.com
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