Related Experiment Video

Updated: May 29, 2026

Rating L-DOPA-Induced Dyskinesias in the Unilaterally 6-OHDA-Lesioned Rat Model of Parkinson's Disease
06:45

Rating L-DOPA-Induced Dyskinesias in the Unilaterally 6-OHDA-Lesioned Rat Model of Parkinson's Disease

Published on: October 4, 2021

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

Simon Rowe1, Amy Evans2, Saeed Kayhanian2,3

  • 1Faculty of Medicine and Health, University of New South Wales, Sydney, Australia.

Movement Disorders : Official Journal of the Movement Disorder Society
|May 28, 2026
PubMed
Abstract

Insights

This Parkinson's disease (PD) gene therapy trial (OXB-102) showed promising safety and potential motor function stabilization in patients. Though terminated early, the study offers insights into novel dopamine gene therapy for PD.

Area of Science:

  • Neurology
  • Gene Therapy
  • Parkinson's Disease Research

Background:

  • SUNRISE-PD investigated OXB-102, a novel lentiviral vector gene therapy for Parkinson's disease (PD).
  • The phase 1/2 trial was dose-escalating but prematurely halted due to sponsor insolvency.
  • This report focuses on the clinical course of 6 enrolled patients.

Purpose of the Study:

  • To provide an investigator-led clinical description of patients in the OXB-102 gene therapy trial.
  • To summarize the safety and potential efficacy of OXB-102 in Parkinson's disease.

Main Methods:

  • Retrospective analysis of individual patient data.
  • Data compilation and summarization from investigator records.
  • Assessment of serious adverse events (SAEs) and clinical outcomes.

Main Results:

  • Six patients received either a low (n=2) or higher (n=4) dose of OXB-102 gene therapy.
  • Eight serious adverse events (SAEs) occurred; only one was definitively linked to the intervention.
  • All SAEs were transient and resolved without lasting effects; motor function showed some stabilization in most patients.

Conclusions:

  • The novel dopamine gene therapy (OXB-102) demonstrated a favorable safety profile in moderate-to-severe Parkinson's disease patients.
  • Preliminary data suggest potential stabilizing effects of OXB-102 on the clinical progression of PD.
  • Further investigation is warranted to confirm the therapeutic potential of this gene therapy approach.
Keywords:
Parkinson's diseaseclinical trialgene therapy

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