CRISPR’s First One-Patient Success Is Becoming a Company: Aurora Launches With $16M to Scale Bespoke Gene Editing
Aurora Therapeutics has launched with $16 million in seed funding from Menlo Ventures to pursue bespoke CRISPR-based therapies for ultra-rare genetic diseases. The company is founded by CRISPR co-inventor Dr. Jennifer Doudna and Dr. Fyodor Urnov, the scientist behind the first single-patient CRISPR therapy developed for an infant known publicly as "Baby KJ." That success provided proof-of-concept for the model Aurora now intends to scale.
Aurora’s founding team includes Edward Kaye as CEO. Kaye previously led Sarepta Therapeutics and Stoke Therapeutics, both known for targeting rare neuromuscular and genetic disorders. He will oversee development of the company's lead program in phenylketonuria (PKU), a rare metabolic disorder caused by mutations in the PAH gene.
PKU is a genetic block in processing a common amino acid, present in almost all protein-containing foods, which can quietly injure the brain unless diet is tightly controlled from birth. Aurora is trying to move from managing that block with lifelong food rules to directly repairing the faulty gene. The company’s program targets a broad range of PKU-causing variants, aiming to treat not just common mutations but also less prevalent, patient-specific ones.
The startup is structured to take advantage of the FDA’s recently introduced “plausible mechanism pathway,” which makes it easier to approve personalized or small-scale therapies when there’s strong biological rationale without requiring large traditional trials that are impossible in ultra-rare genotypes. Instead of treating each rare mutation as a separate project, Aurora plans to group related variants into a single treatment program, shortening development timelines and easing regulatory review.
Aurora’s emergence comes amid growing interest in tailoring gene editing to individual patients. Over the past few years, clinicians and researchers have started to build “N-of-1” medicines, such as custom antisense drugs like milasen for a single child with Batten disease or foundation-backed antisense programs for ultra-rare ALS mutations. Groups such as the n-Lorem Foundation have begun organizing pipelines for these one-patient and nano-rare therapies.
At the same time, a growing set of AI-focused gene editing startups is working on tools and platforms that could turn today’s bespoke interventions into reusable playbooks across many disorders. Our deep dive, 12 Startups Applying AI to Gene Editing: From Custom CRISPR to Zinc-Finger Revivals, maps that landscape, from custom CRISPR editors to AI-guided nuclease design, offering broader context for Aurora’s model.
Topic: Biotech Ventures
