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Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS
Sponsor: n-Lorem Foundation
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.
Official title: An Open-label Multicenter, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
Key Details
Gender
FEMALE
Age Range
49 Years - 49 Years
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2024-11-25
Completion Date
2025-12-16
Last Updated
2026-04-09
Healthy Volunteers
No
Conditions
Interventions
nL-TARD-001
Personalized antisense oligonucleotide
Locations (2)
Columbia University, Irving Medical Center
New York, New York, United States
Houston Methodist
Houston, Texas, United States