Observations on an Open-Label Phase 1/2 Dopamine Gene Therapy Trial (OXB-102/Axo-Lenti-PD) in People with Parkinson's Disease.

Abstract

BACKGROUND: SUNRISE-PD was a dose-escalating, phase 1/2 study investigating a second-generation lentiviral vector gene therapy delivering the genes for dopamine synthesis (OXB-102) to treat Parkinson's disease (PD). The trial was prematurely terminated due to insolvency of the sponsor. OBJECTIVES: The aim was to provide an investigator-led description of the clinical course for the 6 patients enrolled in the OXB-102 trial. METHODS: Individual patient data were extracted, compiled, and summarized from investigator records. RESULTS: Six patients received a low (n = 2) and a higher (n = 4) dose of the OXB-102 gene therapy. There were eight serious adverse events (SAE), of which only one was considered definitely related to the intervention. All SAEs were transient and resolved without sequelae. Efficacy data were limited, but some stabilization of motor function was observed in most of the patients. CONCLUSION: This novel dopamine gene therapy appears safe in patients with moderate-to-severe PD, with some possible stabilizing effects on their clinical course. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.