# Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

> **NCT07226297** · PHASE1,PHASE2 · ENROLLING_BY_INVITATION · sponsor: **n-Lorem Foundation** · enrollment: 1 (estimated)

## Conditions studied

- Charcot-Marie-Tooth Disease Type 2D

## Interventions

- **DRUG:** nL-GARS1-001

## Key facts

- **NCT ID:** NCT07226297
- **Lead sponsor:** n-Lorem Foundation
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ENROLLING_BY_INVITATION
- **Start date:** 2025-10-27
- **Primary completion:** 2027-10
- **Final completion:** 2027-10
- **Target enrollment:** 1 (ESTIMATED)
- **Last updated:** 2025-11-10

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07226297

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07226297, "Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)". Retrieved via AI Analytics 2026-07-06 from https://api.ai-analytics.org/clinical/NCT07226297. Licensed CC0.

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