BOSTON, Mass. — Aurora Therapeutics said it has officially launched with $16 million in seed financing to develop a scalable platform for personalized gene-editing therapies aimed at treating patients with rare genetic diseases.
The company said the funding was led by Menlo Ventures and will support efforts to transform personalized gene editing from one-off, patient-specific breakthroughs into a repeatable model capable of addressing millions of patients with rare mutations. Aurora is focused on developing and commercializing therapies that can be rapidly tailored to many rare genetic variants using modular gene editors and emerging regulatory pathways.
Aurora was founded by Jennifer Doudna, Ph.D., CRISPR co-inventor and Nobel Laureate, and Fyodor Urnov, Ph.D., a leader in translating genome-editing science into human therapeutics. The company is led by Edward M. Kaye, M.D., who brings extensive experience in rare disease drug development.
“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,” said Kaye. “Our team’s deep experience in rare disease drug development positions us to advance this vision with rigor and urgency.”
Aurora’s approach builds on more than a decade of advances in CRISPR technology, including improvements in guide design, AI-generated editors, rapid sequencing, and mutation identification. The company said it is combining these scientific advances with innovations in regulatory strategy and chemistry, manufacturing, and controls to enable parallel development of multiple mutation-specific therapies.
“Since its discovery, CRISPR has offered the promise of treating the root causes of genetic disease, but we lacked a scalable way to bring those therapies to patients with rare mutations,” said Doudna. “By innovating in both clinical development and approval pathways, Aurora is showing the true promise of gene editing for patients who were previously out of reach.”
A central element of Aurora’s model is the use of emerging regulatory frameworks that allow multiple mutations within a single disease to be grouped under a unified development pathway. The company said this umbrella approach is intended to make personalized gene-editing therapies more economically and operationally viable, addressing a longstanding challenge in rare-disease drug development.
“Aurora is opening a new frontier in genetic medicine,” said Johnny Hu, Ph.D., principal at Menlo Ventures. “We believe that the pairing of recent advances in gene editing and AI with a scalable development and regulatory strategy will dramatically expand the number of people who can benefit from gene editing.”
Aurora’s initial program is focused on phenylketonuria, a metabolic disorder caused by a wide range of mutations in the PAH gene that lead to toxic elevations of phenylalanine in the blood. The company said PKU’s well-characterized biology and high unmet medical need align with recently announced regulatory pathways designed to support therapies with a plausible mechanism of action.
“Every week, PKU specialist physicians like me care for individuals with PKU who are unable to consistently maintain safe phenylalanine levels,” said Chet Whitley, Ph.D., M.D., medical director of the PKU Clinic and professor of pediatric genetics and metabolism at the University of Minnesota. “Gene editing has the potential to offer a definitive and durable treatment for PKU and other genetic diseases, and Aurora’s model provides a promising path to extend these therapies to far more patients — not just a limited subset.”
Aurora said it plans to design therapeutics that address multiple PKU-causing mutations from the outset, with the goal of expanding the platform to additional rare genetic diseases over time.
