Gene therapy injection targets Parkinson's in patients with GBA1 mutations

NCT ID NCT07414290

Not yet recruiting Disease control Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-phase trial tests a gene therapy called VGN-R08b in 17 people with Parkinson's disease who have a GBA1 gene mutation. The therapy is injected directly into the brain's fluid spaces to try to improve symptoms. The main goals are to check safety and see if it works.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: VGN-R08b gene therapy
What this could lead to: If successful, this could point toward a new treatment that slows or improves Parkinson's symptoms in people with GBA1 mutations.
What could go wrong: This is an early phase 1/2 trial with only 17 participants, so results may not apply to all patients. The therapy is injected directly into the brain, which carries risks like infection or bleeding.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

GBA1-related Parkinson disease, susceptibility Parkinson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PARKINSON DISEASE (PD)

Contacts and locations

Locations

Xiangya Hospital, Central South University

Changsha, Hunan, 410008, China

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Other studies related to the condition(s) this trial covers.