On February 25, 2026, CobiCure hosted the inaugural event of the CobiCure Impact Series — Children Can’t Wait: Reimagining and Accelerating Pediatric Innovation. More than 90 leaders from across healthcare, biotech, philanthropy, finance, and rare disease advocacy gathered inNew York City for an evening of honest conversation about why the current system leaves children with rare diseases behind, and what it will take to change that.
The evening featured a father’s deeply personal account of losing a child to a disease with no treatment options, a panel discussion on the structural barriers in pediatric drug and device development, and a keynote address on how philanthropic capital and industry investment can work together to create sustainable models for rare disease innovation. Attendees left more informed, more connected, and ready to take action as part of a growing community committed to getting treatments to the children who need them.
Explore each segment of the evening below.
Michele opens the evening withCobiCure’s founding story and mission: to advance treatments for children with rare diseases when the market will not. She describes how CobiCure identifies programs with real scientific promise and takes them forward as a nonprofit, evaluating them on their potential to save lives rather than their potential for commercial return.
Jim explains why Deerfield, a leading healthcare investment firm, built both a for-profit engine and a nonprofit public charity to advance healthcare. He names the scale of the challenge: 30 million Americans have roughly 10,000 rare diseases, most of them children, and they will not benefit from the current wave of biotech and pharmaceutical innovation. He positions CobiCure as the organization that connects the science, funding, and regulatory expertise needed to reach these patients.
Robert shares the story of his son Zachary’s battle with DIPG, the deadliest form of pediatric brain cancer. With a zero percent survival rate and no meaningful treatment advances in four decades, Zachary’s journey represents the human cost of a system that has not invested enough in childhood diseases. Robert connects his family’s experience to the broader reality: only 4% of federal cancer research funding goes to childhood cancers, and commercial incentives are insufficient to drive new treatments for rare pediatric conditions.
The panel explores where the healthcare system fails children with rare diseases, from promising research that stalls at technology transfer because patient populations are too small, to pediatric medical devices that cannot simply be scaled-down versions of adult tools, to reimbursement systems that were not designed for rare conditions. The panelists also discuss emerging solutions: mission-aligned investment capital, cross-institutional collaboration, regulatory reform, and the critical role of organizations like CobiCure in bridging the gaps.
Brienne delivers a keynote on how Royalty Pharma has combined philanthropy with investment to advance treatments for rare diseases, including nearly $50 million in charitable contributions over five years and significant investments in pediatric therapies. She walks through the landmark partnership between the Cystic Fibrosis Foundation and Royalty Pharma as a model for how mission-driven capital can create a virtuous cycle: breakthrough science leads to therapies, therapies generate royalties, royalties become capital, and capital funds the next generation of research.
In the fireside chat, Michele and Brienne dig into practical questions about integrating nonprofit and industry efforts earlier in the development process, overcoming barriers in translational science, and what it takes for a young nonprofit like CobiCure to build sustainable programs.
Michele closes the evening by reinforcing that the evening was about building a community, and that it is just the beginning. She invites attendees and viewers to stay connected as CobiCure continues to grow this work through future events, focused conversations, and new partnerships.
Interested in learning more about CobiCure’s work or future events? Reach out to us or follow CobiCure on LinkedIn to stay up to date.
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