Asset profile
AAV-GAD gene therapy
Gene therapy / editing
Evidence outlook · lead readout
NCT05603312 · Parkinson's Disease
The completed lead evidence is positive, with design limits that temper confidence — see the rationale below for exactly which.
NCT05603312: The trial was randomized, triple-blind, and sham-controlled, but it was very small, with only 5 participants in each AAV-GAD group and 4 in the sham group. Its primary safety outcomes were clinical adverse-event measures, and the posted results included no statistical comparison; the two primary safety findings were not fully informative because one treatment-related adverse event occurred only in the high-dose group while serious adverse events were absent in all groups.
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Upcoming events
(1 total)
Estimated primary-completion dates from ClinicalTrials.gov — registry estimates, never confirmed readout dates; they can move as trials progress.
2029 (1)
October 2029 (1)
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