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nL-LMNB1-001

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

Overview

nL-LMNB1-001 is an antisense oligonucleotide (ASO) drug designed to modulate LMNB1 (lamin B1) expression. It is being developed specifically for treating Autosomal Dominant Leukodystrophy (ADLD) associated with LMNB1 mutation, a fatal central nervous system white matter demyelinating disorder. This personalized treatment represents an example of precision medicine for ultra-rare genetic conditions.

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