drugset / Trial / NCT07177196
Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Retinal Dystrophy
Phase 1/2
Active not recruiting
1 enrolled
n-Lorem Foundation
University of California, San Diego · collab
NaSingle-groupOpen-labelTreatment
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Retinal Dystrophy due to PRPH2 mutation
Timeline
- Start
- 2025-08-28
- Primary completion
- 2027-08
- Completion
- 2027-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nL-PRPH2-001 | Antisense oligonucleotide | — | — |