Stevie Ringel, CEO of Nome Therapeutics, discusses the US Food and Drug Administration’s (FDA) Plausible Mechanism Framework and its effect on rare disease therapy development.
On November 12, 2025, the US Food and Drug Administration (FDA) released the Plausible Mechanism Framework guidance. The purpose of this guidance is to outline recommendations to help developers of individualized therapies generate sufficient clinical safety and efficacy data for the intended use, and that the product can be manufactured to regulatory quality standards. The data is used to support approval or licensure of an individualized therapy for a specific indication. This includes a careful evaluation of the results of nonclinical and clinical data and chemistry, manufacturing, and controls data.
This framework has the opportunity to cut approval timelines, lower costs, and make drug development for rare diseases economically viable.
Mr. Ringel, also a rare disease patient, was told at the age of 17 that he’d go blind and there was no cure for his disease. Over the next 16 years, he realized the science existed but the system couldn’t deliver a treatment efficiently. In response, he built precision medicine business units at Tempus, GeneDx, and Sema4, learning the operational infrastructure of this industry. In October 2025, he launched Nome Therapeutics with an artificial intelligence (AI)-powered orchestration platform that strives to solve the execution problem the recent FDA guidance created.
Historically, genetic medicine has focused on drug development that leads to patient recruitment, which has posed difficulties in meeting enrollment goals and clinical endpoints.
Nome Therapeutics’ model takes this into consideration, using AI systems that scour databases of a whole patient population and find those that will benefit from certain therapies and develop from there. The company currently has five preclinical programs across gene therapies and ASOs in neurology and retinal disease. The objective is to help patients figure out the path forward with blueprints that use AI in addition to science to look for and synthesize all potential options for each patient; then ongoing procurement and program management to develop therapies. This is done with the goal of vetting patients to then move forward to clinical trials.
To learn more about rare disease drug development, visit https://checkrare.com/drug-development/
