"Human cells still know what to do with that protein" made by the lost gene ... but they say their results suggest that a ...

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 ...

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

Vitamin D plays a vital role in the human body, far beyond just keeping bones strong. It helps your muscles move, allows your ...

Epicrispr Biotechnologies CEO Amber Salzman, Ph.D. offers up four imperatives for industry leaders working to develop ...

Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an extra copy of chromosome 21. Down syndrome is also the most common genetic cause ...

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.

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

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

The 28th American Society of Gene & Cell Therapy (ASGCT) meeting kicks off in mid-May in New Orleans. The annual event, which is on pace toward a goal of 8,000 attendees, will be a whirlwind of ...