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# 4 Ways to Better Use CRISPR-Edited Therapies: Article
- URL: https://www.fdaweb.com/4-ways-to-better-use-crispr-edited-therapies-article/
- Published: 2026-04-22T12:00:00.000Z
- Updated: 2026-09-14T13:37:53.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5161020

Two researchers say there are things FDA can do to better use its new plausible mechanism pathway that is expected to increase incentives for drug companies to develop personalized treatments, including gene-editing therapies, for rare disorders. Writing in *Nature*, the researchers say the bespoke nature of personalized gene therapies “has posed a huge obstacle to their spread.”

They say their work has shown that it takes about four years to perform all the tests needed to get FDA approval for a tailored therapy. “This lengthy period might be acceptable for a genetic treatment for a condition such as hemophilia or sickle-cell disease, for which existing medications can keep a person alive for many years,” they write. “But it is much too long for a newborn who has been diagnosed with a severe genetic disease of metabolism, immune system, lungs, or skin, for which treatment in the first year of life is critical. Worse, the full set of studies needed to secure approval to start a clinical trial is expensive, typically costing more than $25 million. That’s too much for drug developers, for a product that will treat just one child.”

Under the proposed new pathway, they say, drug companies would be able to treat many people with different mutations as part of a single clinical trial, as long as participants have the same clinical symptoms. As a result, the researchers say, they foresee that the timeline to get CRISPR therapies into the market could drop to as little as three months, at a cost of less than $250,000 per patient.

The paper lists aspects of the drug development process that the researchers say need to be updated to make the most of the streamlined approval pathway:

- prioritize patient welfare over the need to fulfill all regulatory requirements;
- fund a dedicated agency rapid response team to oversee the pathway;
- engage with patients’ families and advocacy groups early and often;
- prioritize review and approval for severe conditions in which the benefit clearly outweighs the risks; and
- collect as much data as possible, as fast as possible, from early trials to inform ongoing approval decisions.

“Simply approving drugs is not enough to ensure that they make it to patients,” the article concludes. “Targeted federal investment is needed to ensure that the raw materials for drug components are readily available in the U.S…. A therapy that must be manufactured within just a few weeks of diagnosis cannot depend on a globally fragmented supply chain.”