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NPX-NeuroMusc
- Development stage
- Preclinical
- Lead developer
- Neurophoenix
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptide-Drug Conjugates → Peptide Conjugates → Peptides, 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
01
Overview
NPX-NeuroMusc is a preclinical drug candidate developed by Neurophoenix for the treatment of Amyotrophic Lateral Sclerosis (ALS). The therapeutic is a conjugate consisting of a cell-penetrating molecule (CPM) and a phosphorodiamidate morpholino oligomer (PMO). Its mechanism of action involves disrupting the protein-protein interaction between Phosphatase and tensin homolog (PTEN) and the kinases MAST1 and MAST2. This disruption leads to the inactivation of PTEN, which is a key negative regulator of neuronal survival and axonal regeneration. By inhibiting PTEN activity through this specific interaction blockade, NPX-NeuroMusc aims to promote neuroprotection and neuroregeneration in motor neurons affected by ALS. The technology is based on a platform derived from the Rabies virus glycoprotein mechanism for neuronal targeting.
02
Targets
PTEN (Phosphatase and tensin homolog deleted on chromosome ten)MARK1 (Microtubule affinity regulating kinase 1)MARK2 (Microtubule affinity-regulating kinase 2)
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