Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area. 

Casgevy, the world’s first CRISPR gene editing therapy, is the second drug to be accepted onto a managed access scheme via England’s Innovative Medicines Fund, offering a new treatment for patients with transfusion-dependent beta thalassemia.

The company has accelerated a Phase II trial testing the NaV1.8 inhibitor in lumbosacral radiculopathy, and a Phase III trial in diabetic peripheral neuropathy will start this quarter.

Only three of the 10 new therapies approved by the US FDA also have been approved in the European Union, while both of the EU gene therapy approvals followed FDA clearance, a Pink Sheet analysis shows.