Thanks to ELA’s massive investment in research into cerebral ALD, in 2009 the team led by Prof. Patrick Aubourg and Dr. Nathalie Cartier published the first results of a gene therapy trial for cerebral adrenoleukodystrophy conducted in France. This work was then continued internationally by the Bluebird Bio laboratory, which conducted the phase 2/3 trial.
On 21 May 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) gave a favourable opinion for marketing authorisation for gene therapy for the treatment of cerebral adrenoleukodystrophy, and on 21 July 2021, the European Commission (EC) granted marketing authorisation.
This is a historic breakthrough in the treatment of adrenoleukodystrophy.