Praxis Precision medicine announced that its Investigational New Drug Application (IND) for it’s novel early-onset SCN2A epileptic encephalopathy drug (SCN2A-DEE) utilized real-world data from Invitae’s Ciitizen platform. Ciitizen helps accelerate the process of data collection for rare disease therapeutic development. In the development of PRAX-222, the therapeutic in question, Ciitizen compiled the largest dataset of its kind using, in part, electronic health records (EHR) from patients with SCN2A-DEE.
According to Praxis’s co-founder Steven Petrou, PhD, “The comprehensive real-world clinical evidence generated through Invitae’s Ciitizen platform was a critical component to the PRAX-222 IND application, integrating a significant amount of natural history data in a highly efficient manner to help bring PRAX-222 one step closer to SCN2A-DEE patients. In order to make real progress and offer hope to patients living with SCN2A-DEE and their caregivers, it requires a committed ecosystem and a community willing to consider innovative approaches to drug development. We look forward to our continued partnership with Invitae for PRAX-222 and for other precision medicines targeting rare genetic epilepsies with high unmet need.”
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(Source: Yahoo, September 20th, 2022)