Overview

Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

Status:
NOT_YET_RECRUITING

Trial end date:
2027-11-01

Target enrollment:

Participant gender:

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1

Phase:

PHASE1

Details

Lead Sponsor:

n-Lorem Foundation

Collaborator:

Dell Children's Medical Center of Central Texas