Vertex Pharmaceuticals Inc. (NASDAQ:VRTX), a leading biotechnology company specializing in cystic fibrosis (CF) treatments, has been making significant strides in expanding its therapeutic portfolio ...

The bottom line is few companies can match its combination of established leadership in rare diseases and realistic pathways ...

The core reason is the drug's novel mechanism of action and potential to be a viable ... Vertex has hit the ground running with Casgevy, its gene therapy for blood disorders.

January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...

The results suggest that GSK3036656 could become a component of new, simpler treatment regimens for TB, and its novel mechanism of action raises ... sickle cell disease, Casgevy, has been given ...

New York-headquartered Stemline, which licensed global rights to elacestrant from Radius Health in July 2020, said the new drug is the first hormonal therapy with a novel mechanism of action in ...