KJ received the bespoke CRISPR treatment 6.5 months after birth, receiving three infusions of the therapy as of April 2025.

In a world first, scientists used CRISPR to fix a baby's unique genetic mutation. The report offers hope for personalized ...

A newborn boy, KJ, was diagnosed at birth with a rare and potentially fatal genetic disease. He received a novel, on-demand CRISPR therapy in record time.

Drug development takes years, time KJ did not have. But his doctor, Rebecca Ahrens-Nicklas, a metabolic-disease expert at the ...

A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design ...

G ene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has ...

Treated with an individualized gene-editing therapy that corrects mutations directly on the genome, a patient shows improvement from a rare metabolic disease.

Operating loss: $3.5 million, compared to an operating loss of $3.6 million for the second quarter of fiscal 2024.