"Human cells still know what to do with that protein" made by the lost gene ... but they say their results suggest that a gene therapy could someday be a viable option for patients with gout.

Fractyl Health announced a first-in-human trial for RJVA-001, targeting T2D and obesity, pending regulatory approval by 2026. Fractyl Health, Inc. has announced the submission of the first module of ...

Cancer scientists have been working against the clock for decades to outsmart a disease that comes back more powerfully after ...

The commercial success of existing lipid-lowering medications highlights the enormous market potential for effective ...

Biomedical engineers at Duke University have successfully conducted experiments to treat damage caused by heart attacks in nonhuman primates using gene therapy for the first time.

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

Capsida is investigating the root cause of the death, which occurred in the first patient treated in a trial of the company’s ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

In a major step toward treating rare neurological diseases, scientists have used gene editing to correct mutations inside the ...