We are absolutely delighted to hear that the Phase 1/2 Gene Therapy Trial being conducted by AVROBIO has begun! You can read about the trial here. We are immensely grateful to the work of Dr Stephanie Cherqui and her team, all of the clinicians and medical personnel working on this trial, and most of all, the people with cystinosis who are volunteers in this trial. They and their families are pioneers and their generosity incredibly important to the future of cystinosis treatment.
This work could not have begun without the dedicated fundraising of cystinosis supporters, and Cystinosis Research Foundation who invested in the early stages of this research, allowing it to get to this stage. Thank you all.