New Gene Therapy Could Treat 95% of Rare Diseases Currently Without Cures

Medical researchers say they're close to a major breakthrough in treating rare diseases that affect millions of Americans. The new gene therapy technology works like a universal system that doctors can reprogram to target different diseases by changing just one component.

Right now, only about 500 treatments exist for roughly 5% of all known rare diseases. The other 95% of rare diseases have no approved treatments, leaving patients and families with few options.

The costs are crushing for families who do have treatment options. Most rare disease drugs cost between $300,000 and $750,000 per year because they're developed for small patient groups.

Doctors recently used an experimental version of this approach to help a patient known as KJ. His medical team worked across multiple hospitals and compressed years of typical treatment development into just months to create a personalized therapy.

The 21st Century Cures Act has helped speed up approval processes for rare disease treatments, particularly for kidney diseases where FDA-approved options are especially limited.