drugset / Trial / NCT07703462
Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
Phase 1/2
Enrolling by invitation
1 enrolled
n-Lorem Foundation
Thomas Jefferson University · collab
NaSingle-groupOpen-labelTreatment
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
Timeline
- Start
- 2026-12
- Primary completion
- 2028-12
- Completion
- 2028-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nL-TARDB-002 | Unknown | — | — |