Personalized therapies, faster diagnoses, and a renewed and previously unimaginable sense of hope for rare disease families. OpenAI CEO of Applications, Fidji Simo talks not only about how technology is aiding in discovering, diagnosing, and treating diseases, but also about how it has shaped her own health battle.

As scientific and political pressures collide at the FDA, regulatory bottlenecks are intensifying. At this critical and uncertain moment for the rare disease community, we talk about ways to accelerate the path from development to approval, so life-changing therapies more quickly reach the people who need them. Featuring Congressman Jake Auchincloss (D–MA), Former FDA Commissioner Dr. Scott Gottlieb and Former Senator (R-PA) and Rare Disease Advocate Rick Santorum.

The science behind rare disease treatments is advancing at an unprecedented pace. Biogen is sharpening its long-term strategy to lead in the next era of innovation, including ensuring next-generation therapies deliver greater efficacy and durability for patients. CEO, Chris Viehbacher talks about how the company is expanding its global footprint and diversifying its pipeline, all while navigating a rapidly evolving regulatory environment.

We are in the golden age of science and technology, advancements in gene editing and ASO therapies are allowing doctors to deliver targeted, and in some cases one-time, interventions for conditions once considered untreatable. A conversation with Harvard Medical School Professor Dr. Anna Greka and n-Lorem Foundation Chairman & CEO Dr. Stan Crooke about what this means for millions of patients now and in the future.

There’s a pay problem in the quest to help those with rare diseases. The uncomfortable truth behind the breakthroughs is that cures only get funded at scale when the economics work. The hidden kingmaker is not the drug company, it’s insurance. RA Capital Managing Partner Peter Kolchinsky and Aradigm Health CEO Dr. Will Shrank talk about why this happens, what needs to change, and the new models being developed to flip the problem on its head.