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NL-TARD-001
- Development stage
- Phase 2
- Lead developer
- n-Lorem Foundation
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
NL-TARD-001 is an experimental antisense oligonucleotide (ASO) therapy developed by the n-Lorem Foundation. It is designed as a personalized, N-of-1 treatment for individuals with amyotrophic lateral sclerosis (ALS) caused by specific genetic mutations in the TARDBP gene. The drug functions by targeting faulty genetic material (mRNA) associated with the TARDBP gene variant, leading to the reduction of harmful TDP-43 proteins. This allele-selective approach aims to address the underlying genetic cause of ALS, offering a novel strategy compared to existing treatments that primarily focus on slowing disease progression. Clinical trials for NL-TARD-001 are currently in Phase 1/2, focusing on safety and efficacy in a single participant.
- Other names
- NL-TARD-001NL-TARD001NL-TARD 001
02
Targets
TARDBP (TAR DNA-binding protein 43)
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