Publications and Presentations
ASPIRE-FTD Study Update: A Phase 1/2 Clinical Study to Evaluate AVB-101 in FTD with GRN Mutations
Date: Jul 14, 2026
Conference / Journal: AAIC 2026
Author(s): G. Klodowska, J.C. Masdeu, B. Pascual, M. Ratajczak, M. Czarnecki, R.R. Darby, A.G. Murley, J.D. Rohrer, J.B. Rowe, M. Zabek, J.B. Elder, W. Gray, C.M. Powell, J.Y.C. Chan, Y.-G. Huang, D.L. Cooper
Development and translational validation of AVB-406: an intravenous AAV-miRNA targeting MAPT for the treatment of Alzheimer’s disease
Date: Jul 14, 2026
Conference / Journal: AAIC 2026
Author(s): Romain Joubert, Madeline Louise Reilly, Deirdre O’Connor, Gurdeep Kooner, Leonie Sophie Brebner, Deborah Ojutalayo, Martina Esposito Soccoio, Randa Elsayed, Catia Andreassi, Emily Dellow, Bibiana C Mota, Neda Ali Mohammadi, Andrea Martorana, Michael MacDonald, Peter Boyce, Agnieszka M Ule, Carlos J Miranda, Christopher E Shaw, John Isaac, Alex Bloom
Preclinical Development Of AVB-406: An Intravenous AAV-miRNA Achieving Sustained MAPT Knockdown To Treat Alzheimer’s Disease
Date: Jul 13, 2026
Conference / Journal: AAIC 2026
Author(s): Deirdre M. O’Connor, Emily Dellow, Bibiana C. Mota, Neda A. Mohammadi, Anna Ettorre, Zoe Walker, Amrita Abhilash, Gurdeep Kooner, Megan Donnelly, Andrea Martorana, Magda Sztyler, Jaya Kenth-Shah, Josh H. Yeung, Quwam Akinlawon, Guillaume Lo, Douglass Dey, Daniel Cox, Peter Boyce, Romain Joubert, Chris E. Shaw, Alex Bloom, John Isaac, Carlos J. Miranda
Development and translational validation of AVB-406: An intravenous AAV-delivered miRNA targeting MAPT for the treatment of Alzheimer’s disease
Date: May 14, 2026
Conference / Journal: ASGCT 2026
Author(s): Romain Joubert
Manufacture of AVB-406, a BBB–crossing AAV vector for MAPT knockdown in Alzheimer’s disease
Date: May 14, 2026
Conference / Journal: ASGCT 2026
Author(s): Andrea Martorana
Preclinical development of AVB-406: An intravenous AAV-miRNA achieving sustained MAPT knockdown for the treatment of Alzheimer’s disease
Date: May 13, 2026
Conference / Journal: ASGCT 2026
Author(s): Carlos Miranda
Functional Recovery for Photoreceptor Degenerative Diseases with UGX202, a Novel Optogenetic Therapy
Date: May 5, 2026
Conference / Journal: ARVO 2026
Author(s): Lingjie Cui, Zhen Yu, Meng Li, Xueting Luo, Kai Wu
Direct image-guided convective perfusion of the bilateral thalami for gene therapy in frontotemporal dementia: technical note
Date: Dec 19, 2025
Conference / Journal: Journal of Neurosurgery
Author(s): Russell R. Lonser MD,Matthew Rabon BEng, Mark Damante MD, J. Bradley Elder MD, Miroslaw Zabek MD, and David L. Cooper MD
Intrathalamic delivery of adeno-associated viral vector expressing progranulin as gene therapy for GRN-related frontotemporal dementia
Date: Oct 30, 2025
Conference / Journal: Molecular Therapy Methods & Clinical Development
Author(s): Youn Bok Lee, Do Young Lee, Zoe Walker, Joseph Allison, Aleksandra Kaliszewska, Alinda Fernandes, Katharine Y. Zhang, Yalem Bekele, Romain Joubert, Joana Furtado, McQuinn Gumus, Olivier Brock, Ali Bienemann, Steven Gill, Samantha Murray, Nadia Mitchell, Page Bouchard, Alex Bloom, John Isaac, Carlos J. Miranda and Christopher E. Shaw
vMiX™: A novel AAV-based RNA interference platform demonstrating consistent gene silencing efficacy in mouse studies
Date: Oct 7, 2025
Conference / Journal: ESGCT 2025
Author(s): Z Walker
vMiX™ Multiplex: A novel platform for enhanced and versatile miRNA-based gene silencing in neurodegenerative disorders
Date: Oct 7, 2025
Conference / Journal: ESGCT 2025
Author(s): L. S. Brebner
Use of long-read sequencing to iterate and optimise rAAV vector genome design
Date: Oct 7, 2025
Conference / Journal: ESGCT 2025
Author(s): A Abhilash
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