Gene therapy represents one of the most promising frontiers in treating neurodegenerative diseases, offering the potential to address underlying genetic causes rather than just symptoms.
Gene therapy for neurodegenerative diseases involves delivering therapeutic genetic material to target cells to correct or modulate disease-causing genes.
Adeno-associated virus (AAV) vectors are the dominant delivery platform[1]:
CRISPR-Cas9 and related technologies enable precise genome editing:
ASOs modulate gene expression at the RNA level[2]:
GBA mutations are among the most significant genetic risk factors for Parkinson's disease:
LRRK2 mutations cause familial Parkinson's disease:
| Phase | Company | Product | Indication | Status |
|---|---|---|---|---|
| I/II | uniQure | AMT-130 | Huntington's | Recruiting |
| I/II | Prevail | PR001 | Parkinson's (GBA) | Recruiting |
Gene therapy for neurodegeneration remains high-risk but offers substantial potential returns if technical challenges are overcome.
Kaplitt MG, et al. Safety and tolerability of gene therapy with an AAV vector for Parkinson's disease. Lancet Neurol. 2021. ↩︎
Palfi S, et al. Long-term follow-up of AAV gene therapy for Parkinson's disease. Mol Ther. 2022. ↩︎