CTF CEO Annette Bakker Moderates Panel at World Orphan Drug Congress 2025

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.

Tomorrow 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.

Information about the session:

"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

The session will explore how to align unmet medical needs with the right scientific and financial incentives - spotlighting real-world models that turn untapped assets into life-changing therapies.