A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized ...

Ask scientists which gene-editing tool is most needed to advance gene therapy, and they'd probably describe a system that's ...

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia ...

Vertex Pharmaceuticals on Monday reported disappointing first-quarter earnings impacted by weaker-than-expected sales of its ...

May is Cystic Fibrosis Awareness Month - learn about this genetic condition causing thick mucus and organ damage.

Biopharmas have been scaling back on AAV research for sometime now. Find out why and what's on the horizon for drug delivery ...

Advanced cystic fibrosis makes even simple things like walking or showering ... Fundraising efforts have helped jump-start gene therapy that could help patients regardless of mutation. While it likely ...

Vertex Pharmaceuticals missed Wall Street estimates for quarterly results on Monday due to lower-than-expected sales of its ...

Research shows Black cystic fibrosis patients are more likely than their white counterparts to be among the 10% who don't benefit from modulator therapies. Can a gene therapy work no matter the ...

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other ...

Vertex Pharmaceuticals is pressing pause on a phase 1/2 cystic fibrosis (CF) trial over a tolerability issue while reporting ...