Menlo Ventures has made a $16 million bet that the “baby KJ” custom CRISPR therapy success story is repeatable. The funding has enabled CRISPR co-inventor Jennifer Doudna, Ph.D., and baby KJ scientist Fyodor Urnov, Ph.D., to launch Aurora Therapeutics with a vision of treating the long tail of rare diseases.
California-based VC shop Menlo has backed Aurora in the belief that the conditions needed to develop treatments for the rarest of rare diseases, down to conditions that affect a single patient, are now in place. As Menlo sees things, the genetic causes of many of the more than 7,000 rare diseases that affect 350 million people globally are known and correctable. The challenge has been acting on that knowledge.
Menlo named the FDA’s “plausible mechanism pathway,” which offers a route to market for personalized therapies, as one of the changes that mean action is now possible. The VC fund also flagged technologies that could accelerate drug design and testing and support personalized manufacturing as key advances.
Encouraged by the developments, Menlo incubated Aurora, provided seed funding and installed Edward Kaye as CEO. Kaye, the former CEO of Sarepta and Stoke Therapeutics, will lead a team that is pushing a phenylketonuria (PKU) program toward the clinic.
PKU is caused by PAH gene mutations that lead to toxic elevations of phenylalanine. Researchers have found hundreds of gene variants in people with PKU, making the indication a proving ground for Aurora’s ambition to go beyond the relatively common mutations that are typically the focus of rare disease R&D. The biotech plans to address multiple PKU-causing mutations from the outset and add more over time.
Aurora’s approach reflects emerging regulatory support for grouping multiple mutations within a disease into unified development paths. The biotech is betting that grouping variants will make personalized therapies economically and operationally viable. Aurora said its PKU work is supported by preclinical proof-of-concept data and “encouraging” regulatory feedback.