Few fields in medicine are as promising and complex as gene therapy. As researchers race to develop cures for previously ...

The sNDA is supported by data from the phase 3 ENERGIZE and ENERGIZE-T studies, which evaluated mitapivat vs placebo in patients with NTD and TD alpha- or beta-thalassemia, respectively.

Detailed price information for Vertex Pharmaceutic (VRTX-Q) from The Globe and Mail including charting and trades.

Vertex Pharmaceuticals and Orna Therapeutics announce a $4 billion collaboration to develop in vivo gene editing therapies ...

Orna will receive a $65m upfront payment and is eligible to receive potential milestone payments and royalties.

The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle ...

Vertex Pharmaceuticals Inc. will work with a Watertown biotech on new gene-editing therapies for people with sickle cell disease and transfusion-dependent beta thalassemia.

Vertex aims to further bolster its gene-editing therapy pipeline by using Orna's lipid nanoparticle delivery technologies during the three-year collaboration.

Vertex partners with Orna Therapeutics to develop in vivo gene editing treatments for sickle cell disease and beta ...

Orna Therapeutics, through its subsidiary ReNAgade Therapeutics, announced a three-year strategic research collaboration with Vertex ...

Vertex is paying Orna $65 million upfront, in part via convertible note, according to a Jan. 7 release. Orna—which acquired ...

I am Greek and have thalassemia minor. In general, my red blood cells are much smaller than typical ones, and my iron content ...