'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis

Ryan Cross (Endpoint News)

May 8, 2025

Fyodor Urnov had given a version of his speech dozens of times before. Yet as he leaned into the microphone to address the FDA’s top regulators late last year, he felt his heart racing. The agency officials were there to hear about a slow-burning crisis: What do you do when the root cause of a disease is known, the treatment is clear, but drug companies won’t touch it?

It was November 2024, and in a Marriott conference room in Bethesda, MD, Urnov described a tongue-twisting and incredibly rare condition called familial hemophagocytic lymphohistiocytosis, or HLH. Caused by one of several genetic mutations, it overactivates the immune system in young children. Without a stem cell transplant, the children die. Even with one, the prognosis is poor.

“The single thing I need you to understand,” Urnov told the FDA officials in the small, packed room, “is that 40% of infants with inherited HLH do not survive on standard of care.”

Scientists have documented more than 7,000 rare diseases — the vast majority of which affect children, are caused by genetic defects, and have no good treatment. Since the invention of CRISPR gene editing in 2012, the toolbox for manipulating genes, and maybe treating those many diseases, has exploded.

But the number of gene editing cures has not.

Urnov, more than anyone, had been warning that there is a growing gap between what’s possible, and what drug companies find profitable enough to pursue. His alarm has only grown more urgent as biotech startups set up to go after those diseases have made layoffs or shut down. Many of the ones who have survived the sector’s ongoing downturn have pivoted to more common — and more lucrative — conditions. CRISPR’s potential, he argues, is being wasted.

“I have lost all interest in making discoveries with potential,” Urnov told Endpoints News. “The bleak truth is that we don’t need new gene editors, we don’t need new delivery modalities to treat 500 inborn errors of immunity. They’re completely treatable using existing tools and we have multiple capitalized for-profit biotechs that could be treating them — but they’re not.”

For many genetic diseases, Urnov, a University of California, Berkeley professor, believes that science is no longer the limiting factor in making new medicines.

In October 2021, he laid out the biotech industry’s mounting problems in an opinion piece for a scientific journal. He argued that it was technically possible to rapidly develop gene editing therapies for most genetic diseases, and outlined the steps it would take to go from diagnosing the cause of a condition, to making a custom therapy in two months.

“This status quo is not acceptable. Patients dying of N = 1 genetic conditions cannot wait for the for-profit sector to work out its business model,” he wrote.

His solution was to get regulators to start treating CRISPR as a “platform” — industry jargon for a technology that can be used to make treatments for many different diseases — instead of having to treat each use of the technology as a separate drug, with separate preclinical work, trials and regulatory applications, all of which add time and cost.

Credit: Diego Moran/UC Berkeley

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