drugset / Trial / NCT07226297
Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)
Phase 1/2
Enrolling by invitation
1 enrolled
n-Lorem Foundation
The University of Texas Health Science Center, Houston · collab
NaSingle-groupOpen-labelTreatment
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Charcot-Marie-Tooth disease type 2D (CMT2D) due to a pathogenic, de novo deletion mutation in GARS1
Timeline
- Start
- 2025-10-27
- Primary completion
- 2027-10
- Completion
- 2027-10
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nL-GARS1-001 | Antisense oligonucleotide | — | — |