A baby named KJ was born in Philadelphia with an ultra-rare metabolic disorder that, under normal circumstances, would have required an organ transplant or been fatal. Instead, doctors sequenced KJ’s genome and identified a unique mutation. Within six months, they designed and delivered a personalized CRISPR-based therapy — resulting in the first patient to be cured by a bespoke gene-editing treatment.
A few years earlier, a young girl in Boston named Mila suffered from a fatal neurodegenerative disease. In just 10 months, a team of doctors designed, developed and synthesized a one-of-a-kind drug just for her, this time using an antisense oligonucleotide therapy that slowed the progression of her disease.
These are more than medical miracles. They are proof that it is now possible to tailor medicines to a single person’s genetic code. What is missing is a regulatory framework to scale these one-off breakthroughs into a new standard of care.
Globally, more than 50 million children suffer from rare genetic diseases — and nearly one in five will die before the age of five. Even children with the same diagnosis can carry different genetic mutations, meaning a one-size-fits-all drug may be ineffective or impossible to make. The problem is our healthcare system isn’t designed or incentivized to develop drugs for individual patients.
The good news is that science and technology have caught up. We can now sequence a genome for under $100. Artificial intelligence models can design mutation-specific therapies in days and RNA-based treatments can be manufactured in weeks. Operation Warp Speed showed our ability to distribute advanced therapies at scale. And with wearables and digital health tools, we can monitor children’s physiological response in real-time, both before and after treatment.
The FDA’s current approval framework is nearly a century old. It was originally designed to regulate mass-produced drugs for large populations. While there have been reforms since then, most have been incremental and tailored to conventional pharmaceutical development.
The current process treats personalized medicines — especially “N-of-1” therapies — as individual research trials. This approach is time-consuming, expensive and not scalable for the millions of patients who could benefit.
Rare disease doesn’t just affect patients, it drains families and their communities. Parents leave the workforce to become full-time caregivers. Families travel across the country for access to specialists. Experimental treatments, hospital stays, lost wages and uncovered expenses can bankrupt a family before a diagnosis is even confirmed.
In many cases, the child’s condition continues to deteriorate while waiting for a diagnosis or cure. The cumulative damage is incalculable.
Thanks to advances in genomics, AI, synthetic biology and preclinical testing, personalized therapies are becoming more cost-effective and scalable. For example, the FDA’s recent roadmap to phase out mandatory animal testing opens the door to validating therapies using artificial organs or a patient’s own cells — improving safety while cutting time and cost.
America remains a global leader in the core breakthroughs that made Mila and KJ’s treatments possible — from CRISPR gene editing to large language models trained on protein and RNA structures. But that lead is fragile.
The FDA has taken steps in the right direction, including establishing a pathway for personalized CAR-T therapies and issuing draft guidance for ASO drugs. But guidance isn’t enough.
We need a dedicated framework that clarifies the rules, requirements and incentives for those building personalized therapeutics. Without it, researchers and companies will hesitate to invest time and resources into what feels like regulatory guesswork.
As the FDA and the Department of Health and Human Services undergo what appear to be structural changes, this is a rare window to modernize regulation and secure America’s position at the forefront of next-generation medicine. To turn personalized therapies into a new standard of care, the FDA can take a number of key steps.
The agency should create a new regulatory pathway for bespoke therapeutics. N-of-1 drugs can’t be evaluated using the traditional Phase 1-2-3 frameworks. We need an entirely new process-oriented framework that considers families, physicians, payers, regulators and industry alike.
The FDA should establish a dedicated oversight body. A centralized group within the agency should review personalized therapeutics, set transparent safety and ethics standards and ensure rigorous tracking of outcomes. This will build consistency, speed and public trust.
The agency should enable sustainable funding and reimbursement. Cost no longer needs to be the barrier. Clear regulatory rules will unlock investment, while reimbursement frameworks will ensure payers can cover these treatments responsibly. Absent this, safe and effective therapies may never reach patients.
Every week matters for a child with a rare, degenerative condition. Acting now can turn scientific breakthroughs into a public health reality. If we wait, we risk letting outdated regulation stall progress — and watching the rest of the world move forward without us.
The tools are here. The science is ready. All that’s missing is a regulatory system built for the future of medicine that doesn’t treat individualized care as an exception, but as the new standard.
Nessan Bermingham is an operating partner at Khosla Ventures, where he invests in genetic medicines and AI drug discovery and development. He is also the founding CEO of Intellia Therapeutics, the founder and chairman of Korro Bio, and a board member of EveryONE Medicines.