Mukesh Mayani
Senior Director, Global CMC Development Ultragenyx Pharmaceutical Inc.
Seminars
Wednesday 24th February 2027
Fireside Chat: Leveraging FDA Platform Technology Designation Program Learnings to Accelerate Multi-Program Development & Reduce Redundant Studies
10:00 am
- Understanding regulatory interactions and data package requirements for platform designation applications, positioning companies to benefit from streamlined review processes and reduced filing burdens
- Sharing knowledge from companies that have successfully obtained platform designation to reduce redundant development, saving years of process characterization work and millions in development costs
- Navigating applicability of platform approaches for small companies with single assets versus large multi-program portfolios, determining when platform investment delivers meaningful return
Wednesday 24th February 2027
Advancing Empty/Full Capsid Separation Technologies to Increase Potent Product Yield & Minimize Manufacturing Costs
1:45 pm
- Evaluating novel chromatography resins and purification modalities to improve separation of genome containing versus non-genome-containing capsids, reducing the significant product losses that drive up cost per dose
- Implementing emerging analytical technologies including mass photometry, nanomosaic and analytical ultracentrifugation (AUC) for enhanced empty/full characterization to support regulatory filing confidence and process optimization
- Optimizing purification strategies to achieve higher purity levels while maintaining acceptable recovery rates and infectious titer, delivering more doses per batch for improved patient access
