Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation

Phase 1/2 Active not recruiting 1 enrolled n-Lorem Foundation Hawaii Pacific Neuroscience · 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: 2024-10-24
Primary completion: 2026-11
Completion: 2026-11

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	nL-ATN1-002	Antisense oligonucleotide	—	—

Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation

Summary

Timeline

Drugs

Indications