Mobilizing Science, Partnership, And Capability To Develop A Gene Therapy In 14 Months

This webinar tells the extraordinary story of how one family, driven by urgency and guided by pioneering leaders in gene therapy, partnered with Andelyn Biosciences’ AAV experts to develop a treatment for their daughter’s ultra-rare neurodegenerative disorder, NEDAMSS, in just 14 months.

When the Krueger family received their daughter Elly’s devastating diagnosis — caused by an IRF2BPL mutation — they refused to accept inaction. They mobilized a global network of scientists, clinicians, advocates, and manufacturing partners to pursue a bold strategy: developing a gene replacement therapy with the potential to alter Elly’s future. With scientific mentorship from Dr. Kathrin Meyer and the support of rare disease leaders, the team rapidly advanced from concept to clinic, executing development and manufacturing in parallel to compress traditional timelines.

Andelyn Biosciences played a pivotal role, enabling accelerated cGMP vector production through its AAV Curator® platform. Meanwhile, preclinical studies across multiple animal models generated the robust data needed for an IND submission. In just 14 months, Elly received her therapy via intracisterna magna injection—a milestone made possible by extraordinary dedication, strategic risk-taking, and seamless collaboration.

This powerful case study highlights the key enablers of rapid therapeutic development for ultra-rare conditions — patient-led advocacy, manufacturing excellence, scientific execution, and trusted partnerships. The Krueger family’s journey not only offers hope but also provides a replicable model for other rare disease communities determined to transform heartbreak into action.

Key Learning Objectives

• Discover how patient-led advocacy, scientific mentorship, and manufacturing expertise can accelerate gene therapy development through bold, strategic partnerships.
• Learn the critical enablers of speed, including parallel execution, digital modeling, and a robust, platform-based process.
• Explore a replicable framework that rare disease communities can use to move from diagnosis to treatment faster, with practical insights into regulatory, clinical, and manufacturing pathways.