Epilepsy
PRAX-222
Program Detail
Currently in development for patients with early-onset SCN2A-DEE.
PRAX-222 : Introduction
SCN2A-DEE is a monogenic epilepsy disorder caused by variants in the SCN2A gene that encodes the voltage-gated NaV 1.2 sodium channel. SCN2A-DEE is associated with a wide range of phenotypes, treated with complex and burdensome therapeutic regimens. Despite multiple treatments, more than 70% of patients with SCN2A live with uncontrolled seizures. Of those patients, approximately 75% live with severe intellectual disability.
PRAX-222 is an Antisense Oligonucleotide (ASO) that targets the SNC2A gene, and has the potential to be the only disease-modifying treatment for early-onset SCN2A-DEE.
Directly targets the underlying genetic cause of sodium channel dysfunction.
Designed to address both seizure reduction and improvement in development to significantly improve quality of life.
Mechanism of action
SCN2A variants occur when a protein coding sequence causes a strand of mRNA to undergo a missense mutation. SCN2A missense mutations are associated with severe seizure disorders.
PRAX-222 is an antisense oligonucleotide (ASO) designed to down-regulate SCN2A expression in patients with early-onset SCN2A-DEE.
PRAX-222 is an ASO designed to down-regulate SCN2A expression in patients with gain-of-function mutation
Key Upcoming Milestones
Next steps for PRAX-222 Clinical Program
Medical Papers + Presentations
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