Gene therapy
AAV-delivered gene therapies (AADC, GDNF, neurturin, GBA1) and other gene-modifying approaches.
State of the art
No update yet for Gene therapy. An update is a standalone state-of-the-art for the topic — what someone with Parkinson's needs to know about where this approach stands today.
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Observations on an Open‐Label Phase 1/2 Dopamine Gene Therapy Trial (OXB‐102/Axo‐Lenti‐PD) in People with Parkinson's Disease
This is the first complete peer-reviewed publication of the SUNRISE-PD open-label Phase 1/2 data for OXB-102/AXO-Lenti-PD, a lentiviral vector delivering TH, GCH1, and AADC genes to the putamen. Six participants tolerated the therapy without serious drug-related adverse events, and UPDRS-III off scores improved ~40% from baseline; the programme was terminated commercially in 2022 before a sham-controlled efficacy trial could be completed.