Session Information
Date: Tuesday, September 24, 2019
Session Title: Parkinsonisms and Parkinson-Plus
Session Time: 1:45pm-3:15pm
Location: Agora 3 West, Level 3
Objective: This presentation will provide an overview of the design and key objectives of the SUNRISE-PD study.
Background: Parkinson’s disease (PD) is caused in part, by the progressive degeneration of dopaminergic neurons in the substantia nigra. The standard of care, L-dopa, is highly efficacious but long-term use is complicated by motor fluctuations from intermittent stimulation of dopamine receptors and off-target effects. Therefore, a therapy that provides a localized and continuous supply of dopamine to the area of greatest loss in PD, namely the putamen, offers the potential for reduced motor fluctuations and off-target effects for these patients.
Method: AXO-Lenti-PD is a novel gene therapy that delivers three genes which are critical for de novo dopamine biosynthesis, to the putamen, using a high-capacity lentiviral vector. Transduced neuronal cells produce continuous dopamine from endogenous tyrosine in the striatum.
Results: The first-generation construct of this product (known as OXB-101) was found to be well-tolerated throughout administration and long-term follow up with all patients displaying some improvement in the UPDRS part III off score, which was sustained in some patients up to six years. To further increase the potency of this construct, the second-generation product, AXO-Lenti-PD, was developed, utilizing the same genes but in a different configuration that allows for increased dopamine production per genetically modified cell. AXO-Lenti-PD is being investigated in the two-part SUNRISE-PD study, comprised of a dose-ranging phase to confirm the optimal therapeutic dose (building on first-generation product data), followed by a sham-controlled trial to assess the safety and efficacy of the optimal dose from the first part of the study.
Conclusion: AXO-Lenti-PD is a lentiviral gene therapy for the treatment of Parkinson’s disease delivering the three genes necessary for endogenous dopamine synthesis directly to the putamen. It is being evaluated in the ongoing SUNRISE-PD study, comprised of a dose-ranging part to determine the optimal dose and a sham-controlled part to assess the efficacy and safety of that dose.
To cite this abstract in AMA style:
G. Corcoran, P. Korner, J. Wright, Y. Mo, J. Benoit, K. Binley, J. Miskin, N. Tuckwell, D. Zamoryakhin, T. Foltynie, R. Barker, K. Mitrophanous, S. Palfi. The SUNRISE-PD Study, a clinical trial of AXO-LENTI-PD: a CNS-directed gene therapy for the treatment of Parkinson’s Disease [abstract]. Mov Disord. 2019; 34 (suppl 2). https://www.mdsabstracts.org/abstract/the-sunrise-pd-study-a-clinical-trial-of-axo-lenti-pd-a-cns-directed-gene-therapy-for-the-treatment-of-parkinsons-disease/. Accessed June 14, 2025.« Back to 2019 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/the-sunrise-pd-study-a-clinical-trial-of-axo-lenti-pd-a-cns-directed-gene-therapy-for-the-treatment-of-parkinsons-disease/