When we published our 2024 report, children suffering from Leber Congenital Amaurosis 4 (LCA4)—a severe inherited retinal ...

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 ...

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

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

We believe Vertex is in strong financial health, given its robust cash flow generation and low debt. At the end of 2024, Vertex held about $11.2 billion in cash and equivalents.

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

Vertex Pharmaceuticals hit a rough patch in Q1, reporting weaker-than-expected earnings and halting an early mRNA cystic ...

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

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

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

(Reuters) -Vertex Pharmaceuticals missed Wall Street estimates for quarterly results on Monday, due to lower-than-expected ...

A boy with an ultra-rare immune disease which can carry a “death sentence” is living a normal life after taking part in a groundbreaking gene therapy trial. Four-year-old Eisa Hussain now ...