The Children's Tumor Foundation

10/28/2025 | Press release | Archived content

CTF CEO Annette Bakker Leads Panel on Drug Repositioning at World Orphan Drug Congress Europe

The Children's Tumor Foundation (CTF) is proud to share that CEO Annette Bakker, PhD, will moderate an expert panel discussion at the World Orphan Drug Congress Europeon Wednesday, October 29, at 13:45 CET.

Titled "Drug Repositioning - How to Get Shelved Assets that Have Value for the Orphan Drug Value Chain," the session will dive into one of the most promising frontiers in rare disease innovation - breathing new life into previously shelved drug candidates to deliver faster, more cost-effective treatments to patients who urgently need them.

Dr. Bakker will be joined by an exceptional group of leaders driving change across science, investment, and industry:

  • David Tapolczay, Head of Strategy and Licensing, Conduit Pharmaceuticals
  • Christian Hebenstreit, COO & Investor, biotx.ai / Moleculo
  • Anjan Aralihalli, Founder, Raya Therapeutics; Partner, CTI Life Sciences Fund
  • Miika Vikkula, Professor of Human Genetics, Co-Director of the Duve Institute & UCLouvain

Together, they'll explore:

  • The potential of shelved assets- exploring how discontinued or paused drug candidates can be successfully repositioned for rare diseases.
  • A success story- the journey of Gomekli (mirdametinib), a repositioned drug that has become a meaningful treatment option for patients with NF1-related plexiform neurofibromas.
  • CTF's European Drug Discovery Engine Accelerator- a new initiative designed to drive orphan drug discovery through innovative, collaborative models.
  • The investor perspective- examining how public and private entities can support drug repositioning, and why it makes sense for both business and patients.

Drug repositioning isn't just smart science - it's smart strategy. By re-evaluating what's already been discovered, we can cut years off development timelines, reduce costs, and deliver therapies that patients have been waiting for.

This dialogue underscores CTF's leadership at the intersection of science, industry, and policy, and its relentless pursuit to transform research into real-world impact for people living with NF and other rare diseases.

The Children's Tumor Foundation published this content on October 28, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on October 30, 2025 at 20:41 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]