Custom-Made genetic drug targets rare brain disease in first human test
NCT ID NCT07084311
First seen Jul 13, 2026 · Last updated Jul 14, 2026 · Updated 1 time
Summary
This study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disorder. The treatment aims to reduce seizures and improve quality of life by targeting the underlying genetic mutation. Only one participant is enrolled, making this a highly individualized experimental approach.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- nL-ATN1-002 (personalized antisense oligonucleotide)
- What this could lead to
- If successful, this approach could point toward a treatment for DRPLA, a rare and devastating genetic brain disorder with no current cure.
- What could go wrong
- This is an early, single-participant study, so results may not apply to others. The treatment is experimental and risks are unknown.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Hawaii Pacific Neuroscience
Honolulu, Hawaii, 96817, United States
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