The birth of Aurora is built upon a compelling clinical breakthrough: in early 2025, KJ Muldoon received a custom-designed CRISPR gene-editing treatment for CPS1 deficiency.

Within just a few months, the treatment corrected the fatal mutation and significantly improved the patient’s condition. This case became the industry's first successful practice of such a personalized therapy.

However, traditional drug development models struggle to address rare mutations that involve a vast number of variants but extremely few patients per specific type. Aurora’s vision is to transition "on-demand" therapies based on CRISPR gene-editing technology from isolated success stories to a broader population of rare disease patients.

Unlike traditional drug development models, the company plans to leverage a new regulatory path recently introduced by the U.S. Food and Drug Administration (FDA), known as the "plausible mechanism pathway."

This approval framework allows for the evaluation of the safety and efficacy of personalized therapies based on data from a small number of patients. This shortens development cycles and reduces reliance on large-scale randomized controlled trials—a long-standing bottleneck in the field of rare diseases.

Aurora’s core objective is to develop a platform technology for the reproducible and systematic production of personalized gene therapies, specifically targeting rare mutations with minimal patient populations that are difficult to cover in traditional clinical trials.

Dr. Jennifer Doudna stated: "The founding of Aurora marks a turning point for personalized gene editing. We now have the scientific tools and regulatory support to transform isolated successes into a sustainable, parallel development model."

The company plans to utilize AI tools to assist in designing editor components, combined with CRISPR technology, to rapidly generate gene-editing solutions tailored to specific mutations in different patients.

Aurora’s initial focus is centered on rare metabolic diseases such as Phenylketonuria (PKU). PKU is a metabolic disorder caused by various mutations in the PAH gene. Without early intervention, phenylalanine accumulates in the blood, causing brain damage.

Currently, clinical management relies primarily on a strictly protein-restricted diet, and fundamental treatments for different PAH mutations remain very limited.

Aurora plans to use base editing—a precise form of CRISPR—to design standardized processes targeting over a thousand mutations that cause PKU, aiming for a "no mutation left behind" goal. The company will also utilize artificial intelligence to assist in the design of CRISPR editors to enhance the efficiency and accuracy of the therapies.