Encoded is committed to working closely with healthcare professionals to deliver transformative precision gene therapies to patients and their families by addressing the underlying cause of Dravet syndrome and other pediatric central nervous system (CNS) disorders.
We are identifying and optimizing transcription factors and human regulatory elements in relevant organs, starting with the brain, that can be harnessed to generate best- and possibly first-in-class gene therapies with potentially unprecedented gene specificity and cell selectivity.
Our lead asset, ETX101, is a potential one-time, disease-modifying gene regulation therapy for SCN1A+ Dravet syndrome. If you are a doctor who treats patients living with Dravet syndrome, we invite you to view our video below to learn how ETX101 acts at the molecular level to potentially treat the underlying cause of this disease.
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The first participant is expected to be treated in 2022 with ETX101 in our first-in-human investigational
clinical trial. Click here for more details on our clinical trial plans.
Epilepsy Genetic Testing Program
As precision medicine approaches emerge, early genetic diagnosis is vital to enable early intervention and optimal disease management strategies for patients with genetic epilepsies, including SCN1A-related disorders.i
Encoded is proud to be part of Behind the Seizure®, a sponsored, no-charge genetic testing program founded by Invitae and BioMarin. Behind the Seizure makes testing available for healthcare providers to order for any child up to the age of eight who has an unprovoked seizure. This program aims to help provide faster diagnosis for young children with genetic epilepsies.
Research has shown that participants in Behind the Seizure are diagnosed one to two years sooner than historic averages.ii Encoded and program collaborators reported at the 2020 American Epilepsy Society (AES) Annual Meeting that early SCN1A-related disease identification is being facilitated by the Behind the Seizure’s targeted epilepsy gene panel sponsored testing program.
i Truty, R., Patil, N., Sankar, R., Sullivan, J., Millichap, J., Carvill, G., Entezam, A., Esplin, E. D., Fuller, A., Hogue, M., Johnson, B., Khouzam, A., Kobayashi, Y., Lewis, R., Nykamp, K., Riethmaier, D., Westbrook, J., Zeman, M., Nussbaum, R. L., & Aradhya, S. (2019). Possible precision medicine implications from genetic testing using combined detection of sequence and intragenic copy number variants in a large cohort with childhood epilepsy. Epilepsia Open, 4(3), 397–408. https://doi.org/10.1002/epi4.12348
ii Miller, Nicole, et al, “Behind the Seizure: A No-Cost 125-gene Epilepsy Panel for Pediatric Seizure Onset Between 2–4 Years”. Presented at the American Society of Human Genetics Meeting: October 16–20, 2018, San Diego, CA.
Resources and Encoded Contact Information for Healthcare Providers
Visit the Encoded YouTube channel for a three-part educational webinar series for healthcare professionals that provides additional background information on AAV-mediated gene therapy for developmental and epileptic encephalopathies.
If you are interested in learning more about our programs, clinical trials or connecting with a member of our Medical Affairs team, please email firstname.lastname@example.org. By providing your information, you agree to allow Encoded Therapeutics to collect the name and email address provided and to be contacted by Encoded and its partners using this information.