In 2018, a seven-year-old girl named Mila with a fatal neurogenetic disease became the first person in the world to receive a drug tailored to a single patient. The drug was named milasen. In a remarkable collaboration between scientists, physicians, drug developers, foundations and regulators, this novel medicine was created, tested and delivered in just one year from her diagnosis. While Mila would eventually succumb to her condition, milasen suppressed her seizures and improved her quality of life. The 2019 publication of this effort in the New England Journal of Medicine drew international attention as the first example of individualized genomic medicine, setting a precedent and giving new hope to patients with conditions considered too rare to support treatments.

The field has moved quickly since. More than 80 patients have now been treated with over 30 different individualized ASOs. In 2025, the first personalized CRISPR-based therapy was designed, approved, and delivered in six months — a new milestone for the field and proof that the model can extend beyond ASOs. That same year, the FDA released its Plausible Mechanism Framework, establishing a regulatory pathway for ASOs, gene editing, and other individualizable modalities. Together, these developments mark a meaningful shift: the field is no longer asking whether individualized medicines are possible. It is asking how to make them routine.

The N=1 Collaborative was founded in 2021 to build the infrastructure this emerging field required. Its organizing committee brought together scientific leaders from across the globe — among them Dr. Tim Yu, the designer of milasen, and Julia Vitarello, Mila’s mother, who has become one of the field’s most consequential advocates. With founding support from the Oligonucleotide Therapeutics Society N-of-1+ Taskforce and the Chan Zuckerberg Initiative, N1C has grown into a global community dedicated to ensuring individualized medicines reach patients responsibly and at scale.