Krabbe disease Gene Therapy

First Patient dosed and site in UK announced

PassageBio have announced that the first patient has been dosed in the GALax-C study of PBKR03 gene therapy for early infantile Krabbe disease.

Please click on the link to read the full announcement:


The first patient was part of a global phase 1/2 trial. PassageBio have a site in the UK in Manchester University that is active but it is not yet recruiting.

To find out more information about the clinical trial, please click on the link:


Alex - The Leukodystrophy Charity