In 2018, my daughter, Mila, became the first person in the world to receive a medicine designed for one person, an antisense oligonucleotide (ASO) called “milasen.”
Last week, the world received the breaking news that a baby named KJ had received the first personalized CRISPR treatment — one that may save his life. Just as technology is revealing thousands of genetic conditions behind previously unexplained symptoms, Mila and KJ’s stories are proving an entirely new way of treating those who suffer from genetic disease. The new era of individualized medicines is here.
For nearly 80 years, drugs have been developed almost entirely for large populations due to profitability. This model utilizes extensive, costly clinical trials with hundreds of patients, measuring success by average response, often helping only a subset of patients. The traditional approach may work for a few dozen genetic disorders that affect large numbers of people, but certainly doesn’t for 10,000 other small genetic diseases.
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