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

Insulin-producing cells injected into a man with type 1 diabetes have survived for a month so far without the need for immune ...

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.

Most mutations which cause disease by swapping one amino acid out for another do so by making the protein less stable, according to a massive study of human protein variants published in the journal ...

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 ...

You most likely have beta thalassemia minor (one normal gene and one that causes low beta-cell-chain production), which ...

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