Thanks to ELA massive investment in research on cerebral adrenoleukodystrophy (CALD), the team of Pr. Patrick Aubourg and Dr. Nathalie Cartier published in 2009 the first results of a gene therapy trial on cerebral adrenoleukodystrophy conducted in France. This research was extended internationally by the Bluebird Bio laboratory which conducted the phase 2/3 study.
Today, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicine Agency (EMA) adopted a positive opinion recommending marketing authorization for the gene therapy as a treatment in cerebral adrenoleukodystrophy.
This is a major advancement in the treatment of adrenoleukodystrophy.

CP UK