Aurora Therapeutics has officially launched with the mission of transforming personalized gene editing into a scalable solution for millions of patients with rare genetic diseases. The Boston-based company, founded by Nobel Laureate Jennifer Doudna, Ph.D., co-inventor of CRISPR, and Fyodor Urnov, Ph.D., a pioneer in translating genome editing into therapies, has secured $16 million in seed funding from Menlo Ventures. The company aims to overcome the longstanding challenge of treating rare mutations—each unique to an individual—by building a repeatable, systematic platform. Advances in guide RNA design, AI-powered gene editors, and streamlined regulatory and manufacturing strategies are enabling Aurora to move beyond isolated patient breakthroughs toward a model capable of delivering therapies at scale. Edward M. Kaye, M.D., Aurora’s CEO, emphasized the significance of this shift. “We now have the science, tools, and regulatory momentum to go from one-off successes to a sustainable, parallel development model for personalized gene editing,” he said. “Our team’s experience in rare disease drug development allows us to act with both rigor and urgency.” Doudna highlighted the potential of the platform. “CRISPR promised to treat the root causes of genetic disease, but we lacked a way to make that promise real for patients with rare mutations. Aurora is showing how that future can be achieved.” A key innovation is Aurora’s use of umbrella regulatory pathways, which group multiple mutations within a single disease under a unified development strategy. This approach makes it feasible to pursue therapies for a wide range of rare mutations in a cost-effective and operationally efficient way. Aurora’s initial focus is on phenylketonuria (PKU), a rare metabolic disorder caused by hundreds of different mutations in the PAH gene. Without treatment, PKU leads to severe cognitive impairment. Even with strict dietary management, patients often face lifelong challenges with executive function, memory, and social skills. Chet Whitley, M.D., Ph.D., Medical Director of the PKU Clinic at the University of Minnesota, noted the unmet need. “Every week, I see patients struggling to maintain safe phenylalanine levels. Gene editing could offer a one-time, durable cure—and Aurora’s model could bring that to many more people than ever before.” Aurora is leveraging the U.S. FDA’s new plausible mechanism pathway, supported by preclinical data and positive regulatory feedback, to develop therapies targeting multiple PKU mutations from the start, with plans to expand to additional mutations. The company has assembled a leadership team and board of directors with deep expertise in rare diseases, genetic medicine, and biotech development. Menlo Ventures, a leading early-stage investor in digital health and life sciences, expressed strong confidence in Aurora’s vision. “The convergence of CRISPR, AI, and scalable regulatory strategies is unlocking a new era in genetic medicine,” said Johnny Hu, Ph.D., Principal at Menlo Ventures. “We’re excited to back a team that’s making this future a reality.” Aurora Therapeutics is redefining how personalized gene editing is developed and approved, with the goal of making life-changing therapies accessible to patients with rare genetic conditions at a population level.