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PRAX-100
- Development stage
- Preclinical
- Lead developer
- Praxis Precision Medicines
- 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
PRAX-100 is a preclinical antisense oligonucleotide (ASO) candidate being developed by Praxis Precision Medicines in collaboration with The Florey Institute of Neuroscience and Mental Health. It is designed to treat rare genetic neurodevelopmental disorders caused by SCN2A loss-of-function mutations. The drug utilizes Praxis's Solidus ASO platform to modulate the expression of the SCN2A gene, which encodes the alpha subunit of the voltage-gated sodium channel Nav1.2. By addressing the underlying genetic deficiency, PRAX-100 aims to restore normal neuronal function in patients with SCN2A-related disorders, which are often characterized by autism spectrum disorder and intellectual disability.
02
Targets
SCN2A (Voltage-gated sodium channel protein type 2 subunit alpha)
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