Vertex and bluebird compete for sickle cell patients, BioMarin talks about divesting Roctavian, and Sarepta warned that sales of Elevidys will plateau with a narrow label.  

After several rejections by NICE and an appeal by Pfizer, the company has reduced the price for its first-in-class sickle cell disease drug to a level that the health technology assessment institute deems acceptable.

BioNTech and the UK government's alliance to bring personalized cancer immunotherapy to patients is making progress, but the National Health Service has to show it can deliver on clinical trials recruitment.

The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.