Brain gene therapy targets Parkinson's at its source

NCT ID NCT07157345

Recruiting now Disease control Sponsor: Ruijin Hospital Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This early-stage trial tests a new treatment called PDR-001 for Parkinson's disease. It uses a harmless virus to deliver a peptide into a specific brain area, aiming to clear toxic protein clumps that cause the disease. The study will enroll 12 people with early Parkinson's to check safety and see if it might slow the disease.

What this could mean

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

Active substance: PDR-001 (a peptide delivered via gene therapy to clear α-synuclein protein in the brain)
What this could lead to: If successful, this could lead to a treatment that slows or stops Parkinson's disease progression by targeting its root cause, rather than just easing symptoms.
What could go wrong: This is a very early Phase 1 trial with only 12 people, so safety and effectiveness are not yet proven. The gene therapy approach also carries risks like immune reactions or off-target effects.

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.

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

Ruijin hospital
RECRUITING

Shanghai, Shanghai Municipality, 200000, China

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