Agenda Details

Improving AAV Vector Designs: New Concepts for Gene Therapy Translation

Friday, September 6, 2024 08:00 AM - 09:00 AM

Concurrent Session

Chair:

Vincenzo Di Cerbo, PhD, Cell and Gene Therapy Catapult, United Kingdom

Speakers:

Ana Sofia Coroadinha, PhD, iBET - Instituto de Biologia Experimental e Tecnológica, Portugal - Extending AAV genome cargo: delivering large therapeutic genes to treat Duchenne Muscular Dystrophies
Andy Murray, PhD, Sania Tx, United Kingdom - Human-evolved AAV vectors to access specific neural circuits
Ambra Cappelletto, MSc, Purespring Therapeutics, United Kingdom - Kidney Gene Therapy: A Gateway to Treating Glomerular Disease

Adeno-associated viral vectors (AAVs) are the predominant gene delivery modality for in vivo gene therapy. Having demonstrated clinical proof-of-concept through the approval of 8 products in Europe or US, refinement of vector designs is required to improve delivery, targeting, safety, and clinical efficiency. This session will explore vector design strategies to target a range of Indications with an unmet medical need.

Session Objectives:

Learn about novel approaches to improve AAV vector design, including expanding AAV genome cargo capacity, and capsid engineering and evolution.
Explore the path from novel gene therapy concepts to pre-clinical translation for muscular rare disease, kidney diseases, and motoneuron disorders.
Discover how novel AAV platforms can be utilised for the treatment of diseases with large unmet medical need.

Speakers/Moderators

Vincenzo Di Cerbo
Lead Scientist
Cell and Gene Therapy Catapult
Chair

Ambra Cappelletto
Principal Scientist
Purespring Therapeutics
Speakers

Ana Sofia Coroadinha
Head of Lab
iBET - Instituto de Biologia Experimental e Tecnológica
Speakers

Andy Murray
CEO
Sania Therapeutics
Speakers

Session Details