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........

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