Personalized CRISPR therapies could soon reach thousands - here's how

"The FDA's suggested introduction of a 'plausible mechanism pathway' should increase incentives for drug companies to develop personalized treatments, including gene-editing therapies for rare disorders."

"It typically takes about four years to perform all the tests needed to get FDA approval for a tailored therapy, which is too long for newborns diagnosed with severe genetic diseases."

"The full set of studies needed to secure approval to start a clinical trial is expensive, typically costing more than US$25 million, which is prohibitive for many."

"Many genetic conditions could, in theory, be treated with personalized therapies that correct a single genetic mutation in a person's DNA using the CRISPR gene-editing tool."

The FDA's proposed 'plausible mechanism pathway' aims to incentivize drug companies to create personalized genetic therapies, particularly for rare genetic disorders. Over 350 million people globally suffer from more than 5,000 genetic diseases, many of which could potentially be treated with CRISPR gene-editing. However, the bespoke nature of these therapies complicates their development, as each unique guide RNA is treated as a new drug, requiring extensive testing and trials. This lengthy and costly process poses significant challenges, especially for newborns needing urgent treatment.

Read at Nature