We are excited to announce Twelve Below’s recent investment in Nome Bio, an AI-powered platform enabling rare disease patients to develop their own drugs.
Rare diseases affect 1 in 10 Americans and account for over $400 billion in annual medical costs. Although many experts now believe that treatments exist for a meaningful share of these conditions, 95% of rare diseases still lack an FDA-approved therapy. Pharmaceutical companies are rarely incentivized to pursue treatments for such small patient populations, leaving millions of families with a diagnosable disease but no available treatment.
In response, some patients have begun taking matters into their own hands by independently developing drugs to treat themselves. Since 2018, 32 custom drugs have been developed by patients with the help of the FDA’s Expanded Access Pathway, which allows patients to legally develop their own therapies and bypass traditional clinical trials. Yet this DIY process remains fragmented, expensive, and prohibitively complex: patients must coordinate across researchers, manufacturers, consultants, and the FDA. Most families don’t even know this path exists.
Nome is creating a platform for rare disease patients to develop their own drugs. Using AI, Nome evaluates a patient’s genetic data to determine whether their condition is suitable for custom drug development, then coordinates the complex network of researchers, labs, manufacturers, and regulators required to move a treatment from concept to clinic. We believe Nome’s approach represents a transformative new infrastructure layer for rare disease drug development — and personalized medicine at large.
When we met Nome’s founder, Steven Ringel, it was immediately clear that solving this problem is his life’s mission. After being diagnosed with an ultra-rare KIZ gene mutation that causes progressive vision loss, Steven founded a nonprofit to develop a custom gene therapy for his own condition. That insight sparked Nome and its vision to make personalized drug development accessible to every rare disease patient.
Across his decade in genomics and precision medicine — spanning Bain, Illumina, Sema4, and Tempus — Steven has been thinking about how to solve the rare disease problem. With the convergence of key regulatory, technological, and scientific breakthroughs over the past year, Steven set out to build the company that had been chasing him his entire career.
The company’s name honors the 1925 serum run to Nome, Alaska, when dog-sled teams raced antitoxin across frozen tundra to save children in crisis. Nome Bio carries that same spirit of urgency and relentlessness — applying it to the modern challenge of building infrastructure that can deliver life-saving, personalized therapies at scale.
Twelve Below is proud to have led Nome’s $2.7 million Seed round, alongside Refactor Capital and Inflect Health. We’re thrilled to partner with Steven and his exceptional team as they build the infrastructure to make rare disease drug development faster, cheaper, and more accessible than ever before.