Exploration of Neuroprotective Therapy

Table 4. Ongoing clinical trials on gene therapy for PD.

Eligibility criteria	Targeted genes and vectors	Administration process	Safety/Toxicity/Efficacy	Outcome measures	Clinical trials identifier and reference
Phase 1/2a clinical trial of PR001 (LY3884961) in patients with PD with at least one GBA1 mutation	Small molecular GBA gene, AAV9-GBA1	Intra-cisterna infusion followed by methylprednisolone, sirolimus optional	Modified owing to SAEs in the first enrolled participant to receive active treatment	It is anticipated to be finished by 2029, but no data is provided	NCT04127578 [60]
Mild to moderate, and moderate to severe PD patients	GDNF, AAV2-GDNF	Intra-putaminal injection	A robust safety profile	Restoration of dopamine function. To be completed June 2026	NCT04167540

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PD: Parkinson’s disease; GBA: glucocerebrosidase; AAV2-GDNF: adeno-associated virus 2-growth-derived neurotrophic factor; SAEs: serious adverse effects.

Declarations

Author contributions

AOA: Conceptualization, Investigation, Writing—original draft, Writing—review & editing. GOO: Conceptualization, Investigation, Writing—original draft, Writing—review & editing. GAF: Validation, Writing—review & editing. All authors read and approved the submitted version.

Conflicts of interest

The authors declare that they have no conflicts of interest.

Ethical approval

Not applicable.

Consent to participate

Not applicable.

Consent to publication

Not applicable.

Availability of data and materials

Not applicable.

Funding

Not applicable.

Copyright

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Open Exploration maintains a neutral stance on jurisdictional claims in published institutional affiliations and maps. All opinions expressed in this article are the personal views of the author(s) and do not represent the stance of the editorial team or the publisher.

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