A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β ...
You most likely have beta thalassemia minor (one normal gene and one that causes low beta-cell-chain production), which ...
The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle ...
Vertex is paying Orna $65 million upfront, in part via convertible note, according to a Jan. 7 release. Orna—which acquired ...
Orna will receive a $65m upfront payment and is eligible to receive potential milestone payments and royalties.
Few fields in medicine are as promising and complex as gene therapy. As researchers race to develop cures for previously ...
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 Pharmaceuticals and Orna Therapeutics announce a $4 billion collaboration to develop in vivo gene editing therapies ...
The deal is set to help Vertex’s development of next-generation gene editing therapies for sickle cell disease and ...