WinQure Medicine joined leaders from across the rare disease community at the Rare Trials Summit in Boston to discuss how clinical development models need to evolve for rare and ultra-rare therapies.
CEO and Co-Founder Stephanie Manton participated in the Accelerate RARE Working Group alongside Winston Yan from the Broad Institute of MIT and Harvard and the N=1 Collaborative, Nerissa Kreher from Alltrna, Colleen Caleshu from GeneDx, and Dominique Pichard from the International Rett Syndrome Foundation. The group focused on evidence standards for precision therapies, including what constitutes sufficient evidence when conventional randomized trials are not feasible.
Stephanie also joined Casey McPherson, Founder & CEO of AlphaRose Therapeutics, and Andreas Reimann, Co-Founder and Managing Partner at Admedicum, for the session “Ultra-rare, ultra-lean: designing for a viable ROI under 1K patients.” The discussion explored a central question for ultra-rare drug development: what is genuinely required to develop a therapy safely and rigorously, and which elements of traditional development persist primarily because they have become standard practice? When patient populations number in the hundreds, ultra-rare development cannot simply be a smaller version of the traditional biotech model. Development pathways need to be designed for these populations from the outset, while maintaining scientific rigor and patient safety.
Thank you to the Rare Trials Summit organizers and everyone involved for bringing together people committed not only to discussing these challenges, but to working on solutions.