Targeted RNA therapy to restore ATG4B function in ALS

Del01-ATG4B is an exon-skipping oligonucleotide that prevents cryptic exon inclusion in ATG4B mRNA – a disease mechanism identified in ALS patients.

Applications: RNA therapy, Oligonucleotide therapy, ALS, neurodegeneration

Features	Benefits
Broad applicability across sporadic and familial ALS	Potential to address a substantially larger patient population than mutation-specific therapies.
Novel RNA-splicing correction mechanism	Targets a fundamental ALS disease mechanism, potentially slowing neurodegeneration.
Selective restoration of ATG4B expression	Restores normal cellular function while minimising effects on healthy cells
Enhanced CNS delivery using peptide-conjugated ASOs	Enables motor neuron targeting, sustained CNS exposure, and potentially less invasive administration
Mechanistically validated target with promising preclinical results	Provides clear rationale and route for therapeutic development