Annette Bakker

Opinion author

Annette Bakker is a scientist and changemaker redefining how we unlock the value of shelved assets. As CEO of the Children’s Tumor Foundation (CTF), her approach is refreshingly direct: unite top talent, break down silos, and push relentlessly for outcomes. Under her leadership, the Foundation has delivered two NF1 drug approvals, two venture-philanthropy investments, launched platform-basket trials, and progressed a working treatment for NF2-SWN. She has spearheaded novel gene therapy programs and established a preclinical hub to accelerate clinical-ready assets. Her leadership in Europe’s IMI2-funded platform trials has set a new global standard for rare-disease collaboration.

Drawing on two decades in oncology R&D at Janssen (J&J), Siena Biotech, Yale, and the Institut de Myologie, Annette now co-leads global initiatives designed to replicate the mirdametinib repositioning success.

Her model is redefining how nonprofits, pharma, investors, academia, patients, and regulators collaborate to turn “dead” programs into medically and commercially valuable treatments. In recognition of her impact for and with patients, she was named Officer in the Order of Leopold by the King of Belgium.

Had Pfizer’s Freda Lewis-Hall not stepped in, SpringWorks’ rare disease treatment may never have reached patients. Pharmas can act now to help find the next Gomekli.
No longer on the periphery of the life sciences market, nonprofit groups are bridging the gaps in drug discovery and development.