Custom drug targets rare blindness in One-Patient trial
NCT ID NCT07177196
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study tests a custom-made drug called an antisense oligonucleotide, designed specifically for one person with retinal dystrophy caused by a PRPH2 gene mutation. The drug aims to correct the genetic error and potentially slow vision loss. The trial involves only one participant and focuses mainly on safety, while also checking for any changes in vision and eye structure.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
personalized antisense oligonucleotide (nL-PRPH2-001)
What this could lead to
If it works, this could point toward a treatment for retinal dystrophy caused by PRPH2 mutations, potentially slowing vision loss.
What could go wrong
This is a very early, single-person trial, so results may not apply to others. The treatment is experimental and safety is the main focus; it may not improve vision.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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University of California San Diego
San Diego, California, 92093, United States