Eliana was just three months old when she was diagnosed with ADA-SCID, a rare immune disorder that can be deadly if left untreated. Now 11 years old, she is one of five children born with this genetic condition who have been healthy for more than a decade, thanks to a gene therapy developed by UCLA and University College London.

Published today in the journal Blood , long-term follow-up data from 62 children treated with this gene therapy between 2012 and 2019 show a 95% success rate, with no serious complications reported. Armed with this clinical data from the largest and longest study for a gene therapy of its kind, the researchers are aiming for the treatment to receive FDA approval within the next two to three years.

“These results are what we hoped for when we first began dev

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