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; the rationale below says 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.
How the evidence outlook works ↗
Featured trials
The studies most instrumental to this drug's clinical progression, individually graded on design and result. Pick an indication; Research unlocks the trials, endpoints, key results and grades.
Competitive landscape
60 competitors in this universe: 0 direct rivals (same mechanism and indication), 0 same-class, 60 same-indication; 39 further along in development.
| Drug | Stage | Modality | Mechanism | Evidence signal | Reported efficacygraded featured trials |
|---|---|---|---|---|---|
| AAV-GAD Gene Therapy: this drug | Phase 2 | Gene therapy / editing | Glutamic acid decarboxylase (GAD) gene delivered to the subthalamic nucleus | ||
| Amantadine ER (Gocovri / ADS-5102) | Approved | Small molecule | NMDA receptor | ||
| Apomorphine (Apokyn) | Approved | Small molecule | dopamine | ||
| Apomorphine Sublingual Film (Kynmobi) | Approved | Small molecule | dopamine | ||
| Crexont (IPX203) | Approved | Small molecule | dopamine precursor | ||
| Entacapone (Comtess) | Approved | Small molecule | COMT | ||
| Foslevodopa / Foscarbidopa (Vyalev) | Approved | Small molecule | dopamine precursor | ||
| Inbrija (CVT-301) | Approved | Small molecule | dopamine precursor | ||
| Istradefylline (Nourianz) | Approved | Small molecule | adenosine A2A | ||
| Levodopa-Carbidopa Intestinal Gel (Duopa) | Approved | Small molecule | dopamine precursor | ||
| Opicapone (Ongentys) | Approved | Small molecule | COMT, PDE4 | ||
| Parcopa | Approved | Small molecule | dopamine precursor | ||
| Pimavanserin (Nuplazid) | Approved | Small molecule | 5-HT2A/2C receptor, Serotonin 5-HT2A receptor (antagonist / inverse agonist) | ||
| Pramipexole (Sifrol/Mirapex) | Approved | Small molecule | Dopamine D2/D3 receptors | ||
| Pramipexole ER (Mirapex ER) | Approved | Small molecule | Dopamine D2 / D3 receptors | ||
| Rasagiline (Azilect) | Approved | Small molecule | MAO-B |
…and 45 more. Research shows the complete landscape.
Unlock the full comparison
Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.
Upcoming events
(1 total)
Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.
2029 (1)
October 2029 (1)
What to watch
All registered studies
Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.