Personalized CRISPR therapies could soon reach thousands — here’s how

In February, the FDA proposed a new approach to developing personalized genetic therapies, aiming to make it easier for drug companies to create treatments for rare genetic disorders. This change could significantly speed up the process of getting CRISPR gene-editing therapies to patients, especially newborns with severe conditions. Currently, each personalized therapy requires extensive testing, which is costly and time-consuming. The FDA’s new ‘plausible mechanism pathway’ would allow multiple patients with similar symptoms to be treated in a single trial, reducing the time and cost involved. This could mean that life-saving treatments reach patients much faster, potentially within three months and at a fraction of the current cost. The first CRISPR therapy would undergo full testing, but subsequent therapies would need only minimal testing if they are similar, making personalized treatments more accessible. QUESTION: How might the FDA’s new approach to personalized genetic therapies change the future of medicine for rare genetic disorders?