Matthew Herper closing the event and inviting us for cocktails and mocktails at the rooftop terrace. #STATBreakthrough

We need systems that connect. We have tools never had before, and can intervene earlier and at scale. Science keeps on moving but we need trustworthy tools that patients trust. Did they study this in people like me? Can I afford it? Will it be available to me? #STATBreakthrough

Closing remaks by Dana Spencer from Genentech Ai is like an adolescent child; you can give it some rules but it is going to test the boundaries. What feels natural for us might be overwhelming for patients. We need to earn trust. Trust happens at the speed of understanding. #STATBreakthrough

A very long audience question that is more like a monologue. People are starting to shout. It is the end of the day and cocktails on the rooftop terrace might be in the near future. #STATBreakthrough

TL: many biomedical innovations such as CRISPR come from the US. HFL: the ethics of sham surgery trials: they might be needed if we do not know if the treatment works. The UniQure example was handled terribly. www.statnews.com/2026/03/02/u... #STATBreakthrough

UniQure plans to seek approval...

Audience question: should research into drug development and gene therapy not be socialized? Companies might not want to focus on those. YW: it is also an incentive: those people will be very motivated to pay! TL: in many countries those patients take would not be treated. #STATBreakthrough

YW: it is a roller coaster. Representing a therapeutic company I worry about the recent and continuing changes at FDA. Lack of consistency. HFL: We don’t want all drugs approved, but we also don’t want good drugs to not be approved over quibbles about study design. #STATBreakthrough

Next: The Rough Math of Rare Disease Panel with * Ted Love, BIO; Global Blood Therapeutics * Holly Gernandez Lynch, Uni of Pennsylvania * Yael Weiss, Mahzi Therapeutics * Jason Mast, moderator TL: some very recent FDA views go against loosening rules to allow more innovation. #STATBreakthrough

AD: these tools only get better if we use it every day. But human in the loop is important! At BioMarin, we award crowns (King or Queen of AI) at the end of the day if your work contributed to patient health. #STATBreakthrough

AD: many rare diseases do not have their own ICD (International Classification of Diseases) code or are binned together, making it hard to analyze data. LLMs are good at reading human language so can look in more detail. #STATBreakthrough