Our goal at Encoded is the development of viral gene therapies that provide life-changing benefits to individuals with genetic disorders. With recent advancements in sequencing technologies and gene-based medicines, symptomatic treatment and chronic disease management are no longer enough. Instead, we believe in creating treatment options that provide lasting benefit to dramatically improve lives.
Our diverse and impactful gene therapy pipeline addresses devastating genetic and acquired disorders spanning multiple disease pathways, including: neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular disease.
Taking on the Challenge of Dravet Syndrome
Dravet syndrome is a catastrophic form of intractable epilepsy that begins in infancy and occurs in approximately 1 in 16,000 births worldwide. Current therapies typically reduce seizures, but do not address the underlying cause of the disorder.
The majority of Dravet syndrome cases are caused by loss-of-function mutations in the SCN1A gene, which reduce its function by 50%. This leads to uncontrolled seizures, ataxia, and significant developmental delays, as well as a 15-20% mortality rate before adulthood.
Using the Encoded platform, we have developed an AAV vector capable of restoring SCN1A to normal expression levels specifically within the affected cell type, GABAergic inhibitory neurons.
In preclinical studies1, a single dose of our optimized viral vector has dramatically reduced seizures and extended survival in a well-established mouse model of Dravet syndrome. The Encoded gene therapy for the treatment of Dravet syndrome is currently progressing towards clinical development.
1 Young AN, Tanenhaus A, Chen M, McLaughlin J, Belle A, Li J, Lin W, Place K, Rodriguez D, White K, Kearney J, Tagliatela SM. A GABA-Selective AAV Vector-Based Approach to Up-Regulate Endogenous Scn1a Expression Reverses Key Phenotypes in a Mouse Model of Dravet Syndrome. Oral presentation at: American Society of Gene & Cell Therapy Annual Meeting; 2019 Apr 29 – May 2; Washington DC, US.