Platform-Based Gene Therapy Manufacturing Strategies for Scale-Up

As the cell and gene therapy sector accelerates, developers face mounting pressure to move quickly from discovery to clinical trials—and ultimately to commercial launch. With over 3,000 gene therapy clinical trials currently underway worldwide, the demand for scalable, high-quality viral vector manufacturing has never been greater.

For many organizations, outsourcing to a CDMO is a critical step. But not all manufacturing approaches are created equally. Increasingly, platform-based solutions are emerging as a key strategy to de-risk development, standardize processes, and enable scalability.

Understanding Platform-Based Manufacturing

Platform-based manufacturing refers to the use of templated, pre-qualified processes that can be adapted across multiple programs. These platforms typically include:

• GMP-ready workflows that reduce development time and regulatory risk.
• Scalable production systems, from adherent cultures to large-scale suspension bioreactors.
• Standardized analytics and purification strategies that ensure consistent quality.

Why Platforms Matter More Than Ever

Recent research highlights several reasons why platform-based approaches are gaining traction:

• Novel capsid types and AI-designed viral vectors are becoming more common, requiring adaptable manufacturing strategies. A Nature article explores how artificial intelligence is being used to optimize capsid design for better targeting and reduced immunogenicity.
• Standardized analytics and purification methods help reduce variability and improve regulatory readiness. Studies published in ScienceDirect emphasize the importance of harmonized QC frameworks for viral vector production.
• Scalability is essential as therapies move into late-stage trials. Platform-based systems allow for smoother tech transfer and faster scale-up, reducing delays and cost overruns.
  

Evaluating CDMO Partners: What to Look For

When selecting a CDMO, decision-makers should consider:

• Does the CDMO offer platform-based solutions that can support both early-stage and commercial-scale production?
• Are their processes GMP-ready and regulatory-aligned?
• Can they accommodate novel vector designs and evolving therapeutic modalities?
• Do they provide transparent timelines and risk mitigation strategies?

CDMOs like AGC Biologics have developed proprietary platforms—such as BravoAAV™ and ProntoLVV™—to support these needs, offering flexible solutions that can scale with a therapy’s lifecycle.

Platforms like BravoAAV™ and ProntoLVV™ are examples of how CDMOs are responding to these needs, but the core value lies in the approach, not just the product. AGC Biologics’ recent case study reveals how platform-based solutions expedite the path to GMP manufacturing by streamlining development with a plug-and-play system.

Built on a foundation of shared process steps, common equipment, and standardized reagents, AGC Biologics’ own platforms offer flexibility to adapt to diverse Genes of Interest (GOIs) and various AAV serotypes. Notably, our robust LVV services are well-suited to support the unique requirements of gene-modified cell therapy development and manufacturing.  Access the full case study.

Building for the Future

As gene therapy continues to expand into new indications and patient populations, the ability to scale efficiently and reliably will be a defining factor in success. Platform-based manufacturing offers a proven path forward—one that balances speed, flexibility, and quality.

For therapy developers, partnering with a CDMO that embraces this model can be the difference between a promising candidate and a commercially viable product.

Lean into the Experts

CDMOs can offer invaluable knowledge and assistance to drug sponsors looking to bring a product to market. Not only can a CDMO provide optimized development and platform manufacturing systems as discussed above, but they can also guide your product through the necessary regulatory hurdles for commercial approval. AGC Biologics has one of the most experienced teams in the cell and gene industry, with 10 commercial approvals, a 30-year track record of quality, reliability, and regulatory success, and boasts one of the only sites in the world to achieve FDA & EMA approvals in the cell and gene space.

Reach out to our team to discuss how AGC Biologics can help you achieve your project goals.

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