N=1 Collaborative

Stories

FDA creates new approval pathway for gene therapies tailored to individual rare disease patients

Coordinating platform for individualized therapy development

For decades, the FDA required all drugs to prove effectiveness through controlled trials with sufficient sample sizes, a standard that made sense for common diseases but created an impossible barrier for ultra-rare conditions. On February 23, 2026, the FDA issued draft guidance for a new standard—the 'plausible mechanism' framework. Developers of gene-editing and ribonucleic acid (RNA) therapies could win full approval by showing that their treatment targets the root genetic cause, engages it, and improves outcomes compared to the disease's natural course.

Updated May 29

The six-month cure: gene editing goes bespoke

New Capabilities

Building open ecosystem for personalized medicines

In August 2024, KJ Muldoon was born with a death sentence encoded in his DNA—a single broken letter among three billion that left his body unable to process protein. Six months later, he walked out of Children's Hospital of Philadelphia, cured by a gene-editing therapy designed, manufactured, approved, and delivered in 180 days.

Updated May 19