ePoster

BrainVector: A comprehensive platform for pre-clinical development of AAV-based gene therapies for brain diseases

Sverre Grødem, Guro H. Vatne, Nils-Anders Labba, Anders Malthe-Sørenssen, Marianne Fyhn
FENS Forum 2024(2024)
Messe Wien Exhibition & Congress Center, Vienna, Austria

Conference

FENS Forum 2024

Messe Wien Exhibition & Congress Center, Vienna, Austria

Resources

Authors & Affiliations

Sverre Grødem, Guro H. Vatne, Nils-Anders Labba, Anders Malthe-Sørenssen, Marianne Fyhn

Abstract

Technological advances have made Gene therapy a promising therapeutic strategy for a wide range of neurodegenerative diseases. Adeno-associated viruses (AAVs) have demonstrated significant potential as safe and effective vectors for the delivery of therapeutic genes to the CNS. Despite the tremendous promises of AAV-based gene therapies, the pathway to develop these therapies from bench to clinic remains complex and arduous. In response to these challenges, we introduce the BrainVector project, a comprehensive cross-disciplinary platform that streamlines the pre-clinical development of AAV-based gene therapies for brain diseases. BrainVector integrates genetic engineering, neuroscience, bioinformatics, and translational medicine to design, optimize, and test AAV vectors in pre-clinical models. Key components of BrainVector include a sophisticated computational tool to optimize therapeutic gene sequences and promoters, high throughput screening systems in neuronal cultures and stem cell-derived in vitro models for initial evaluations, as well as a pre-clinical assessment pipeline utilizing advanced mouse disease models. By offering an integrated approach to gene therapy development, BrainVector has the potential to expedite the pre-clinical development process, reduce associated costs, and expand the availability and accessibility of these innovative therapies for patients suffering from brain diseases. On this poster, we share the blueprint of BrainVector, and the prospects for its future implementation in the rapid development of AAV-based gene therapies for brain diseases.

Unique ID: fens-24/brainvector-comprehensive-platform-f41865ed