Recent Presentations
American Society for Gene & Cell Therapy (ASGCT) - May 2026
285. Next-generation Gene Therapy for Duchenne Muscular Dystrophy: Enhancing Safety and Expanding Patient Eligibility. Juliette Hordeaux, Ali Ramezani, Bo Xing, Jinlong Jian, Anna Lynch, Hongwei Yu, Elizabeth L. Buza, Peter Bell, James M. Wilson.
386. Preclinical Development of a Next-Generation, Cisterna-Magna-Delivered Gene Therapy for Spinal Muscular Atrophy Type 1. Juliette Hordeaux, Gourav Roy Choudhury, Puya Aravand, Hongwei Yu, Jing-Xu Zhu, Elizabeth L. Buza, Peter Bell, James M. Wilson.
Congresso INAME - November 2025
Gene Therapy for SMA Type 1: History, R&D on Next-Generation Therapy, and the Challenge of Affordability. James M. Wilson.
Foundation for Angelman Syndrome Therapeutics (FAST) Global Science Summit - November 2025
MVX-220: A Gene Replacement Therapy for Angelman Syndrome. Elizabeth Berry-Kravis & James M. Wilson.
CDKL5 Forum - October 2025
Sir Colin Blakemore Memorial Lecture: Gene Therapy as a Disruptive Technology. James M. Wilson.
European Society for Gene & Cell Therapy (ESGCT) - October 2025
P0325. Nonclinical Efficacy of MVX-220, an AAVhu68-based Investigational Gene Therapy, in a Mouse Model of Angelman Syndrome (AS). Heather Born, Richard Fetterly, Allyson Berent, Jennifer Panagoulias, Juliette Hordeaux, James M. Wilson.
INV74. Lessons learned from 35 years of in vivo gene therapy research. James M. Wilson.
American Society for Gene & Cell Therapy (ASGCT) - May 2025
410. Position-Specific Impact of RGD Motif in AAV Capsid Variants on Muscle Tissue Targeting and Integrin Binding Affinity. Rosemary L. Meggersee, Payal Grover, Swapnil Shewale, R. Jason Lamontagne, Juliette Hordeaux, Jenny A. Greig, James M. Wilson.
946. Prevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) 8 and 9 in a Specific Pathogen-Free (SPF) Colony of Cynomolgus Macaques. Marcela Salazar Werner, Jenny Greig, Surina Boyd, Qiuyue Qin, Chelsea Wallace, Jessica Chichester, James M. Wilson.
1025. Gene Replacement Therapy for Barth Syndrome Using a Novel Cardiotropic Capsid. Juliette Hordeaux, Nicholas Roberts, Yingxian Chen, Swapnil Shewale, Ali Ramezani, Clarissa Shoffler, Dina Abbasian, Christopher Petucci, James M. Wilson.
2011. Single Cell Analysis of AAV9 Transduction in a Patient that Succumbed to Acute Respiratory Distress Syndrome Following Systemic High Dose Gene Therapy. Juliette Hordeaux, R. Jason Lamontagne, Peter Bell, Sushobhana Bandyopadhyay, Jing-Xu Zhu, Hongwei Yu, Yanqing Zhu, Darcy Reil, Juan Putra, Richard Horgan, James M. Wilson, Terence R. Flotte.