This week on The Genetics Podcast, Patrick is joined by Stevie Ringel, co-founder of Nome and founder of the Kizuna Foundation. They discuss Stevie's own ultra-rare disease diagnosis and shaped his path to founding Kizuna Foundation and Nome, how Nome's AI agents help scientists navigate the operational complexity of small-batch drug development, the technical and business model advantages underpinning Nome's accuracy, and what it will take to build a sustainable funding model for ultra-rare disease.
Show Notes
0:00 Intro to The Genetics Podcast
00:59 Welcome to Stevie
01:37 Stevie's inherited retinal disease (IRD) diagnosis and subsequent path into genomics
03:16 Why Stevie founded Kizuna Foundation and why ultra-rare drug development is so operationally complex
06:27 The origin story of Nome and using AI to automate the operational work
10:09 The inspiration for the name “Nome” and who the company is built to serve
12:44 The biggest blockers to program speed
15:07 How AI and scale can bring down the cost of gene therapy manufacturing
18:01 FDA signals and global regulatory competition
19:33 Priority review vouchers and why Nome stays out of molecule IP
20:33 Nome's AI and review process for patient reports and its expansion to health systems
25:04 Nome's agent architecture and the data behind its accuracy
28:17 Why delivery remains gene therapy's biggest bottleneck and approaches for solving it
31:34 The case for a new capital model in rare disease drug development
33:25 What’s next for Nome as they advance preclinical programs
34:25 Nome’s focus on process excellence across therapeutic modalities
36:34 Closing remarks
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