One-Time brain injection targets genetic form of Parkinson's
NCT ID NCT07685444
First seen Jul 06, 2026 · Last updated Jul 07, 2026 · Updated 1 time
Summary
This early-stage trial tests a single injection of a gene therapy called LY-N001, given directly into the brain fluid of people aged 30 to 70 with moderate-to-advanced Parkinson's disease who carry a GBA1 gene mutation. The main goal is to check safety and side effects, but researchers will also look for signs of improvement in movement and other symptoms. Only 18 participants will be enrolled, and they will be followed for both short-term and long-term effects.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- LY-N001 injection (gene therapy)
- What this could lead to
- If successful, this could point toward a one-time gene therapy to slow or improve motor symptoms in a specific genetic form of Parkinson's disease.
- What could go wrong
- This is a very early, small trial (18 people) focused on safety. The treatment involves a brain injection, which carries risks like infection or bleeding. It may not improve symptoms or could cause side effects.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, 230022, China
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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