64137 - Parallel Session 1: CMC Challenges with Ultra Rare Diseases

Bringing treatment to those afflicted with an ultra-rare disease is complex. These products are encumbered with challenges ranging from high development costs with a limited ROI, to issues with executing and interpreting clinical trials with unusually small patient populations. Furthermore, the cost to generate the quantities of product required to support an approvable CMC package can be prohibitive for many organizations. 




This session will address ways in which companies have tackled the CMC and regulatory challenges when bringing these life changing therapies to patients in need.




Session Speakers:




Manufacturing Challenges Limiting the Access to ATMPs 

Ralf Altenburger, F. Hoffmann-La Roche Ltd.




Development of a Single Patient CRISPR Therapeutic 

Richard Horgan, Cure Rare Disease




Additional Panelists:




Emmanuel Adu-Gyamfi, CBER, FDA

Becky Schweighardt, Grace Science LLC