Aurora Therapeutics CRISPR Rare Disease Strategy Leverages New FDA Pathway

Can a treatment designed for just one infant change the entire medical industry? In February 2025, a baby named KJ received a personalized gene-editing therapy created in just six months to fix a rare genetic mutation. It saved his life. Now, Aurora Therapeutics, a startup cofounded by Nobel laureate and CRISPR pioneer Jennifer Doudna, aims to scale this bespoke approach to help thousands of others suffering from rare diseases.

Aurora Therapeutics CRISPR Rare Disease Focus via FDA Plausible Mechanism

The company is banking on a new regulatory framework called the "plausible mechanism pathway." Announced by FDA officials last fall, this program allows for the approval of personalized treatments based on data from a handful of patients rather than thousands. According to Marty Makary and Vinay Prasad in the New England Journal of Medicine, this bypasses the traditional roadblock where rare diseases couldn't recruit enough trial participants.

Targeting PKU with Precise Base Editing

Aurora's initial target is phenylketonuria (PKU), a metabolic disorder affecting roughly 13,500 people in the US. CEO Edward Kaye explains that they'll use base editing—a more precise form of CRISPR—to create standardized yet customizable therapies. Fyodor Urnov, cofounder and scientist at UC Berkeley, emphasizes their mission: "No mutation left behind."