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 ...

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

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using CRISPR-Cas9 in combination with AAV6 vectors can trigger inflammatory and ...

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 ...

A trio of common amino acids may hold the key to unlocking far more powerful gene therapies. Researchers found that adding ...

Two papers published in The New England Journal of Medicine highlight the potential of gene editing for treatment of sickle ...

For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the most frustrating bottlenecks in gene therapy has been deceptively simple: the ...

Genetic disorders occur due to alterations in the primary genetic material, deoxyribonucleic acid (DNA), of an organism. Transthyretin amyloidosis (ATTR) is a progressive disorder involving amyloid ...