Publications and Presentations
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
Development and qualification of a robust in vitro relative potency assay using thaw-and-use cells for AVB-101
Date: Oct 7, 2025
Conference / Journal: ESGCT 2025
Author(s): R Lyth
ASPIRE-FTD Study Update: A Phase 1/2 Clinical Study to Evaluate AVB-101 in FTD with GRN Mutations
Date: Jul 30, 2025
Conference / Journal: AAIC 2025
Author(s): Yige Huang
Diagnosis of Frontotemporal Dementia is Often in Non-Specialist Settings and Complicated by Non-FTD Dementia and Psychiatric Diagnoses: Patient and Provider Findings from Analysis of a Large US Real-World Dataset
Date: Jul 28, 2025
Conference / Journal: AAIC 2025
Author(s): D.L.Cooper
Direct Image Guided Convective Perfusion of the Bilateral Thalami Offers a Consistent Approach to CNS Dosing: First-in-Human Experience with Gene Therapy for Frontotemporal Dementia
Date: May 16, 2025
Conference / Journal: ASCGT 2025
Author(s): D.L.Cooper
Effective knockdown of ATXN2 following intracerebroventricular administration of AVB-205 construct in BAC-ATXN2-Q72 transgenic mice
Date: Dec 7, 2024
Conference / Journal: International Symposium on ALS/MND
Author(s): P.M. Coan
A Translational Assay for Ataxin-2 Related Drug Development Products
Date: Dec 7, 2024
Conference / Journal: International Symposium on ALS/MND
Author(s): B. Rougemont
Effective SOD1 targeting with vMiX™, an innovative AAV-based RNA interference platform
Date: Dec 7, 2024
Conference / Journal: International Symposium on ALS/MND
Author(s): L. Reilly
vMiX , an innovative AAV-based RNA interference platform: from in vitro development to in vivo validation by targeting genes involved in ALS
Date: Oct 24, 2024
Conference / Journal: ESGCT 2024
Author(s): R. Joubert