NCT07177196

Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Retinal Dystrophy

An Open-label Single Center Study of an Individualized Antisense Oligonucleotide Treatment for Retinal Dystrophy Associated With a Pathogenic PRPH2 Variant

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: n-Lorem Foundation · Academic / Other
• Sex: Female
• Age: —
• Healthy volunteers: Not accepted

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

Detailed description

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with Retinal Dystrophy due to PRPH2 mutation

Conditions

• Retinal Dystrophy

Interventions

Type	Name	Description
DRUG	nL-PRPH2-001	Personalized antisense oligonucleotide

Timeline

• Start date: 2025-08-28
• Primary completion: 2027-08-01
• Completion: 2027-08-01
• First posted: 2025-09-16
• Last updated: 2025-09-16

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study