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.

The CEOs of bluebird bio and CRISPR Therapeutics hope to inspire investor confidence that their gene-based therapies for sickle cell disease and beta thalassemia will be widely reimbursed in the US and EU.