Children’s Tumor Foundation CEO Annette Bakker to speak on unleashing the life-changing potential of shelved assets at this year’s World Orphan Drug Congress.
At the World Orphan Drug Congress 2025, Dr. Annette Bakker, CEO of the Children’s Tumor Foundation, will moderate a keynote panel on a critical challenge – and opportunity – in rare disease research: how to unlock the full potential of drug repositioning through smarter collaboration and strategic alignment – including ways to better incentivize companies to get involved.
Repositioning offers a faster, more cost-effective path to treatments – especially in rare diseases where patients can’t afford to wait. But realizing this promise requires more than just scientific insight; it takes coordinated action across sectors: academia, nonprofits, investors, and industry.
A recent example: mirdametinib, now FDA-approved for NF1. CTF played a strategic role in advancing this repositioned therapy in collaboration with SpringWorks Therapeutics – demonstrating how previously shelved assets can be reactivated to meet urgent patient needs.
“Repositioning Drugs for Rare Diseases: Unleashing the Potential of Shelved Assets in Pharma and Biotech.” — Wednesday, April 23, 2025 at 9:05 AM ET
Moderator:
- Annette Bakker, CEO, Children’s Tumor Foundation
Panelists:
- Freda Lewis-Hall, Former Chief Patient Officer And Executive Vice President, Pfizer
- Andrew Lo, Charles E. and Susan T. Harris Professor, MIT Sloan School of Management
- Tanisha Carino, Visiting Fellow, Duke-Margolis Institute for Health Policy
- Rena Conti, Associate Professor, Markets, Public Policy, and Law, Boston University
Update: April 24, 2025 – View this recording of the session:
