Accelerating Gene Therapy Development Through Targeted CMC and AAV Platform Optimization

Poster promoting targeted CMC development for AAV platforms, with downloadable PDF link and Pharmaron branding at ASGCT 2025.

Poster Authors:

Amy Hawksworth, Paul Young, Louise Winstanley, Gareth Berry, Shandel Pariag

Pharmaron Gene Therapy | Estuary Commerce Park, Speke, Liverpool, L24 8RB, United Kingdom

Flexible, Data-Driven CMC Strategies for AAV Advancement

Efficient and targeted CMC development is critical for advancing gene therapies through regulatory pathways and into the clinic. Pharmaron’s AAV platform streamlines this process using a manufacturing feasibility assessment that delivers actionable upstream and downstream insights in just 6 weeks.

Utilizing their multi-serotype AAV platform, Pharmaron can assess product fit through scalable bioreactor systems (Ambr?250) and plug-and-play downstream tools. The platform is designed to flex across various serotypes and gene constructs, allowing rapid customisation or fast-tracking to GMP if the product performs optimally.

Key Features of Pharmaron’s AAV Platform Process:

  1. Upstream Screening with Ambr?250 Vessels: Pharmaron evaluates genome and viral titres, transfection efficiency, and batch consistency across AAV serotypes using triple transfection in HEK293 suspension cells.
  2. Downstream Scalability and Toolbox Flexibility: A modular purification strategy using affinity and polishing chromatography yields >70% recovery across diverse serotypes. Empty/full capsid separation is benchmarked early via ion exchange (IEX) profiling.
  3. Early-Stage Optimization or Acceleration: The feasibility study outputs critical data to identify whether targeted process customization is needed, or if the product can proceed directly to GMP manufacture.
  4. DoE & HTPD for Rapid Optimization: When improvement is necessary, high-throughput platforms and statistical design of experiments (DoE) are deployed for data-driven process refinement.

Understand how Pharmaron’s targeted CMC approach enables faster, more confident AAV clinical readiness. Download the full poster presented at ASGCT 2025 to evaluate your path from feasibility to GMP manufacturing.