Bluebird bio's $1.8 Million Setback on Gene Therapy: Is It a Growing Sign of Rejection of Reimbursement Profit Models?

"Some bad news about a $1.8 million gene therapy echoed across the Atlantic Ocean this week and carries implications for California’s state stem cell agency, which also is engaged in developing so-called “one-and-done” therapies. 

" The matter involves bluebird bio of Cambridge, Mass., and its proposed Zynteglo therapy for the rare blood disorder beta thalassemia. The European Union last week refused to go along with bluebird’s price for the treatment -- $1.8 million. 

"Bluebird, which spells its name in all lowercase, said it would fold up its European operations...." "This news (about bluebird) plus Orchard's departure demonstrates urgency around affordability that needs to be addressed, especially when likely sickle cell disease therapies come to fruition,” said an observer who has followed CIRM (the state stem cell agency)  for years, but declined to be identified publicly.

“The technology is similar to proven therapies in X-linked SCID and beta thalassemia that are languishing. Serious consideration needs to take place around utilizing ‘march-in’ rights built into CIRM's IP (intellectual property) policies to force the provision of these therapies in an affordable manner...."

Read more on the new platform for the California Stem Cell Report about the growing problems with "one-and-done" therapies, bringing them to the marketplace and whether public funding models need to be changed significantly.