drugset
drugset / Trial / NCT06706388

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

NCT06706388 ↗

Phase 1/2 Active not recruiting 1 enrolled n-Lorem Foundation Columbia University · collab
NaSingle-groupOpen-labelTreatment

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

Timeline

Start
2024-02-21
Primary completion
2027-02
Completion
2027-02

Drugs

EvaluationDrugModalityDoseRoute
Subject nL-ATN1-002 Antisense oligonucleotide — —

Indications

dentatorubral-pallidoluysian atrophy