Advancement and Challenges in AAV mediated Gene Therapy: Navigating Capsid Design, Formulation, and Delivery

Adeno-associated virus (AAV), a non-pathogenic single-stranded DNA virus that transduces both dividing and non-dividing cells, has emerged as a leading gene therapy platform. It persists primarily as an episode with a low insertional mutagenesis risk and elicits a comparatively mild
immune response. Eleven AAV-based therapies have been approved by the FDA Yet significant challenges limit how far the platform can go. Pre-existing neutralizing antibodies and treatment-induced immunogenicity restricts eligibility and blocks re-dosing, native packaging capacity is limited to ~4.7 kb, production still suffers from low yield and high empty-to-full capsid ratios, and finished products require stable formulations that needs to withstand ultra-low-temperature (≤ -60°C) storage, and controlled freeze-thaw without loss of potency. This presentation will discuss AAV's basic biology and structure, capsid engineering, manufacturing, purification, and
formulation advances and approaches to overcome these limitations.

Chair:
-Dr. Mathew Cherian PhD, FIP, USA.

Co-moderator:
Dr. Richard Ohene-Darkoh, Pharmaceutical Country Manager, Liberia.

Panellists:
-Dr. Subhro Guhathakurta PhD, Review Editor Frontiers in Molecular Neuroscience, USA.
-Dr. Sandeep Nema PhD, Ironwood Pharmaceuticals, USA.

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