Ty Sperle says he felt "insane shock" after learning he'd been cured of a rare genetic disease through a clinical trial using a new gene-editing treatment.

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

"Gene editing takes centre stage in FDA’s new rare disease approval pathway" was originally created and published by ...

After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.

To be the first person in the world cured of chronic granulomatous disease, a rare genetic condition, feels a little surreal ...

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.

Imagine if, instead of delivering a leaflet individually to each home, a postal worker just had to give one to a volunteer on each block, who then photocopied it and handed out copies to neighbours.

Scientists are testing an entirely new way to fight heart disease: a gene-editing treatment that might offer a one-time fix for high cholesterol. It’s very early stage research, tried in only a few ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

Gene therapy and gene editing are marvels of modern biotechnology, providing revolutionary techniques for fixing genetic abnormalities. Gene therapy introduces functional genetic material to ...

Scientists achieved the smell by editing genes in tomatoes responsible for their aroma to make them smell similar to popcorn.