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QRL-201
- Development stage
- Phase 1
- Lead developer
- QurAlis
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
QRL-201 is a first-in-class splice-switching antisense oligonucleotide (ASO) developed to restore the expression of STATHMIN-2 (STMN2), a protein critical for neuronal repair and axonal stability. In amyotrophic lateral sclerosis (ALS) and other motor neuron diseases, loss of nuclear TDP-43 leads to mis-splicing and decreased expression of STATHMIN-2, contributing to neurodegeneration. QRL-201 modulates splicing of the STMN2 mRNA, preventing aberrant intron inclusion and thereby restoring functional protein levels. The drug is administered intrathecally and is currently being evaluated in Phase 1 clinical trials for ALS, with the primary objective to assess safety and tolerability[1][3][4][5][6][7].
02
Targets
STMN2 (Stathmin-2)
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