CRISPR tries to crack the rare-disease problem

Aurora Therapeutics launches with $16M, aiming to turn CRISPR gene editing into a repeatable model for rare disease treatments

13 Jan 2026

Aurora Therapeutics entered the biotech scene with a familiar promise and a sharper focus. Backed by $16 million in seed funding led by Menlo Ventures, the startup wants to solve one of gene editing’s most stubborn problems: scale.

CRISPR has dazzled scientists for over a decade. Turning that brilliance into approved therapies, especially for rare diseases, has been far harder. Small patient populations strain traditional drug economics, often leaving viable treatments stuck in the lab. Aurora thinks the industry has been approaching the problem the wrong way.

Its first target is phenylketonuria, or PKU, a rare inherited metabolic disorder. But PKU is less the destination than the proving ground. Aurora’s real ambition is to build a repeatable development platform that can be reused across many genetic diseases, cutting time, cost, and complexity with each new program.

The company plans to reuse core elements of CRISPR development and take advantage of emerging FDA pathways. In some cases, regulators are willing to consider shared mechanisms of action across related conditions. That shift could make it easier to extend one successful therapy into several others without starting from scratch each time.

Aurora’s founding team includes Jennifer Doudna, a co-inventor of CRISPR Cas9 and one of the technology’s most influential advocates. For years, Doudna has argued that gene editing will fall short of its promise unless new business and regulatory models emerge alongside the science.

The timing may be right. Recent approvals of CRISPR-based treatments for sickle cell disease by Vertex and CRISPR Therapeutics marked a turning point. Gene editing is no longer just experimental. It is a real therapeutic category with commercial legs.

Still, the road ahead is demanding. Regulators require long-term safety data, and personalized therapies remain difficult to standardize. Yet many in the industry see Aurora as part of a broader shift in mindset.

In gene editing, discovery is no longer enough. Scaling is the new measure of success. If Aurora can pull it off, rare disease patients could finally see science catch up with possibility.