The US Food and Drug Administration (FDA) is investigating the need for regulatory action on Bluebird's Skysona.
Skysona is a gene therapy approved by the FDA in 2022 that halts a childhood-onset fatal brain disease. Long-term follow up of 67 children has shown that the therapy has been associated with an increased risk of developing blood cancer (see BioNews 1260).
See Reuters for more information.
