Objective: To characterize the global clinical landscape and temporal evolution of disease modifying cell and gene therapy trials in Parkinson’s disease (PD), including modality distribution, delivery strategies, regional patterns, and current activity.

Background: Across 98 unique interventional, disease modifying PD trials employing cell or gene modalities, activity has progressed from an initial program in 1997 to a clear surge in 2023–2025 (10, 12, and 12 starts, respectively), with continued initiations in early 2026 (see Figure 1).

Method: We filtered trials to include interventional designs with disease modifying intent. Modalities were classified as cell therapy, viral vector gene therapy, or non viral/oligonucleotide gene modulation; routes, vectors, regions, and patient segment labels were extracted from protocol text and normalized where feasible. Data were obtained and curated with Citeline | TrialTrove. Yearly starts and status fields were used to quantify growth and activity. M365 Copilot was used for text editing, data harmonization, and formatting of tables and figures.

Results: Cell therapy predominates (66/98), alongside viral vector gene therapy (32/98); non viral/oligonucleotide programs were rare. Twenty four studies are currently open and eight planned, indicating sustained momentum (see Table 2). Intracerebral/intraputaminal administration – typical for AAV and iPSC derived products – is the dominant delivery paradigm; intrathecal administration appears chiefly with non viral oligonucleotide approaches. Regionally, the Americas and Asia contribute the greatest number of advanced stage, surgically delivered programs – Americas (30 cell; 19 viral vector), Asia (28 cell; 9 viral vector) – whereas Europe shows a smaller but diversifying portfolio (see Table 1).

Conclusion: Over a 26+ year interval, the PD pipeline has expanded and diversified, with cell therapy currently exceeding gene therapy in unique trial count even as gene modulating approaches broaden. The recent upswing in trial initiations, widening geographic participation, and consolidation of brain delivered modalities collectively signal a pivotal transition toward biological restoration as an emerging treatment paradigm in PD (see Figure 1, Table 1, and Table 2).

Figure 1

Table 2

Table 1

References: 1. Winston G, Kharas N, Svenningsson P (2025). Gene therapy for Parkinson’s disease: trials and technical advances. The Lancet Neurology (Personal View).
2. Cell Press (Molecular Therapy) (2025). Recent developments in gene therapy for Parkinson’s disease. Molecular Therapy.
3. Grote J, Patel N, Bates C, Parmar MS (2024). From lab bench to hope: a review of gene therapies in clinical trials for Parkinson’s disease and challenges. Neurological Sciences 45:4699–4710.
4. CiRA, Kyoto University (2025). Treating Parkinson’s Disease with Cell Therapy (clinical trial experience using iPSC‑derived dopamine neurons). CiRA Reporter (News & Events).
5. TreeFrog Therapeutics (2025). Overview of Parkinson’s Disease Clinical Trial Landscape. Corporate analysis article.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/reframing-treatment-paradigms-the-rise-of-cell-and-gene-therapies-in-parkinsons-disease/