Leveraging Platform And Process Characterization Data To Accelerate CGT Validation And Commercialization

By Sanjay Nilapwar (AbbVie), Marcos Sousa, Bernd Tscheschke (Bayer), Kayla Garrett , Matthew Stebbins (Bristol MyersSquibb), Christopher Boyd (Pharmaron), Gina Donovan (Regeneron), Sonia Bedi (Formerly of Regeneron),Silvia Gomez (Bayer (Viralgen)), César Trigueros (Bayer (Viralgen)), Ane Quesada (Bayer (Viralgen)), Vineet Kumar, Kevin Stanson (Roche) ,Yuanli Song (Sanofi), Brian Mullan (Yposkesi), Kathleen O’Hagan (BioPhorum)

Streamlining process development and validation for cell and gene therapy (CGT) products is essential to ensure the timely delivery of therapies to patients while maintaining sustainable product pipelines. Leveraging process development data and standardized platforms can significantly reduce program timelines by supporting efficient manufacturing and validation activities.

This poster highlights work conducted by 16 member companies of the BioPhorum ATMP Phorum, resulting in a deliverable that outlines requirements and strategies for leveraging prior knowledge and process platforms in CGT development. The paper provides key considerations for utilizing process characterization to minimize the testing and studies needed during process validation for drug substance (DS) and drug product (DP) manufacturing, with an emphasis on aligning with regulatory guidelines.

While many recommendations are particularly relevant to gene therapies (GTs), the broader principles can inform strategies for cell therapies (CTs), including developing tailored manufacturing platforms and leveraging approaches for batch or continuous manufacturing processes as appropriate.