[{"orgOrder":0,"company":"Invitae","sponsor":"Praxis Bioresearch","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Invitae\u2019s Real-World Ciitizen Data Utilized in Praxis Precision Medicines\u2019 PRAX-222 IND Filing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Invitae","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Invitae Partners with BridgeBio Pharma to Harness Genetic Insights for the Discovery of Rare Disease Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery"}]
Find Clinical Drug Pipeline Developments & Deals by Invitae
The goal of the collaboration is to generate new insights focused on genetic modifiers and the discovery of novel therapeutic targets for rare diseases and other unmet medical needs.
PRAX-222 is an ASO designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations.