Intellia Therapeutics has reported the first-ever phase 3 results with an in vivo gene-editing therapy, hereditary angioedema ...

The company's treatment for hereditary angioedema reduced attacks by 87% vs. placebo in a Phase 3 trial, clearing the way for ...

Intellia also separately announced positive topline data today from the Phase 3 HAELO clinical trial of lonvo-z in HAE. The trial met its primary and all key secondary endpoints, demonstrating that a ...

CRISPR functions as a programmable genetic memory system derived from bacterial immune defenses against viral infections. Guide RNA allows for rapid multiplexed targeting compared to older ...

Two Philadelphia doctors who led the groundbreaking gene-editing treatment of baby boy from Delaware County are included ...

Sidra Medicine, a member of Qatar Foundation, has been qualified by Vertex Pharmaceuticals as one of a of a limited number of ...

Scientists have taken an important step toward a gene therapy that could one day turn off the extra genetic material that ...

For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...

Clustered regularly interspaced short palindromic repeats (CRISPR) refer to the small fragments of viral DNA that are stored by the bacteria as a part of their defense mechanism. CRISPR–Cas9 is a ...

Qatar has taken a landmark step in precision medicine after Sidra Medicine became the first hospital in the country — and one ...