In the meantime current gene therapies remain expensive and challenging to scale up because each patient’s batch of cells has ...

Improving sickle cell care by expanding treatment options, advancing new therapies and amplifying the voices of people with ...

Vertex said sickle cell patients had not yet received infusions of its gene therapy Casgevy. That’s now changed, as the ...

Kendric Cromer, 12, is among the first patients to be treated with gene therapy just approved by the F.D.A. that many other ...

It’s been almost a year since the Food and Drug Administration approved the first genetic treatments for sickle cell disease.

The 12-month price targets, analyzed by analysts, offer insights with an average target of $508.38, a high estimate of ...

Vertex Pharmaceuticals Incorporated (Nasdaq ... [exa-cel]) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).

If you’re on the fence about investing in Vertex Pharmaceuticals Incorporated or Regeneron ... of cystic fibrosis (CF), and one approved therapy that treats severe sickle cell disease (SCD) and ...