Doudna’s Aurora Launches with $16 Million to Industrialize Personalized Genetic Therapies

Rare Daily Staff

Aurora Therapeutics, a company seeking to transform personalized gene editing from a one-patient breakthrough into a scalable model capable of bringing therapies to millions of people with rare diseases, unveiled itself today.

Menlo Ventures backed the company with $16 million in seed financing to create what it calls the first platform for treating rare genetic mutations that have historically been impossible to address at scale.

Aurora was founded by Jennifer Doudna, CRISPR co-inventor and Nobel laureate, and Fyodor Urnov, scientific director of the Innovative Genomics Institute. Edward Kaye, who most recently served as CEO of Stoke Therapeutics, will lead the company as CEO.

Aurora is working to shift personalized gene editing from isolated breakthroughs to a platform capable of benefiting people with rare mutations at the population scale.

A key component of Aurora’s model is its use of emerging regulatory frameworks that support grouping multiple mutations within a disease into unified development paths. This umbrella approach is designed to make personalized therapies economically and operationally viable, addressing a longstanding gap in rare-disease drug development.

The company’s initial program focuses on phenylketonuria, or PKU, a metabolic disorder caused by a wide range of mutations in the PAH gene that lead to toxic elevations of phenylalanine in the blood. Without early treatment and careful monitoring, PKU can lead to impaired brain development in children and adolescents. Even when early and strict disease control enables near-normal IQ, elevated phenylalanine levels can continue to negatively affect cognitive functions, such as executive function, memory, and social skills, throughout life. For this reason, PKU requires lifelong management.

“Aurora’s launch signals a turning point for personalized gene editing. We now have the science, tools, and regulatory tailwinds needed to move from isolated success stories to a sustainable way of developing many therapies in parallel,” Kaye said. “Our team’s deep experience in rare disease drug development positions us to advance this vision with rigor and urgency.”

Photo: Nobel laureate Jennifer Doudna