Overview

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

Status:
ACTIVE_NOT_RECRUITING

Trial end date:
2027-08-01

Target enrollment:

Participant gender:

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

Phase:

PHASE1

Details

Lead Sponsor:

n-Lorem Foundation

Collaborator:

University of California, San Diego