Gene Editing Has Struggled To Go Commercial. This Nobel Laureate Has A $1 Billion Plan To Fix That.

^{"Jennifer Doudna" by Duncan Hull for the Royal Society via Wikimedia Commons licensed under CC by SA 3.0}

Soon after KJ Muldoon was born in August 2024, he was lethargic and wouldn’t eat. His worried doctors realized his ammonia levels were scarily high. Further tests showed the infant had a rare metabolic disorder—the kind of diagnosis that’s often a death sentence.

A team of researchers from Crispr pioneer Jennifer Doudna’s Innovative Genomics Institute and doctors from the Children’s Hospital of Philadelphia and Penn Medicine began sprinting to create a custom treatment to fix Baby KJ’s DNA, using Crispr-based gene editing. Within just six months, they designed the therapy, got lightning-fast approval from the FDA and, in collaboration with life sciences giant Danaher, manufactured it. Baby KJ received his first infusion on February 25, 2025, and today is a healthy one and a-half year old. 

It was perhaps the most significant milestone yet for the relatively new field of gene editing, which Doudna helped originate more than a decade ago. But even she was floored by how quickly the team...