GT-AADC is a recombinant, adeno-associated virus, containing the human cDNA encoding the AADC enzyme that is currently being developed for the treatment of AADC deficiency. The goal of this therapeutic approach is to increase the amount of AADC in target areas of the central nervous system, thus increasing the production of critical neurotransmitters and subsequently improving neurological function. There are no approved therapies for patients with AADC deficiency.

GT-AADC has been assessed in two clinical studies. Together, these two studies include a total of 18 children with severe AADC deficiency. Patients have shown clinically meaningful and statistically significant increases in motor function over time as measured by two separate motor scales, the Peabody Developmental Motor Scale, second edition (PDMS-2) and Alberta Infant Motor Scale (AIMS), as well as the achievement of clinically meaningful motor development milestones.

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