The company’s CEO Cynthia Pussinen joined the company last year and talked to Scrip about her plans to advance a cell therapy for type 1 diabetes.

As drug pricing continues its ascent among hot button issues, the multi-million-dollar price tags for one-and-done advanced therapies, their justification and reimbursement all remain unaligned.

Supporting ‘n of 1’ gene therapy development, the playbook from a Foundation for the National Institutes of Health-backed consortium aims to help investigators who are not traditional product sponsors prepare for meetings with US FDA and IND submissions.

Dust has barely settled around the uncertainty stirred up by the Federal Trade Commission’s view of the Amgen/Horizon merger and now the US regulator is taking another surprising action.