Encoded Therapeutics is a South Francisco, Calif.-based precision gene therapy company developing precision gene therapies for a broad range of severe genetic disorders. Led by co-founder and chief executive officer Kartik Ramamoorthi, Ph.D., the company is advancing its lead asset, ETX101, for the treatment of SCN1A+ Dravet Syndrome. Encoded leverages a discovery engine that combines biological and computational approaches to identify and screen human DNA regulatory elements at high throughput. ETX101 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the U.S. FDA for SCN1A+ Dravet Syndrome. The company intends to use proceeds from the Series D to conduct clinical trial activities, including a natural history study and first-in-human trials for ETX101, and to progress its pipeline of gene therapies for additional pediatric CNS disorders. The article reports the company raised $135M in Series D financing. Encoded Therapeutics develops precision gene therapies that aim for cell-type selectivity, potency, and the ability to modulate expression of endogenous genes. The company leverages a proprietary platform and initially uses clinically‑validated adeno-associated viral (AAV) vectors. It is focused on four core research areas: neurocircuitry disorders, liver and metabolic disease, neurodegeneration and cardiovascular disease. Encoded’s lead program targets Dravet syndrome, a severe genetic disorder characterized by uncontrolled seizures, ataxia, developmental delays and elevated risk of SUDEP. The company raised $104M in a Series C and intends to use the proceeds to advance its Dravet program, push its preclinical pipeline and expand platform-enabled programs for severe genetic disorders. Encoded was incubated by Illumina Accelerator, seeded by Venrock and ARCH Venture Partners, and is led by co-founder and CEO Kartik Ramamoorthi, Ph.D.

Total raised

$239M

Funding rounds

2

Latest round

Series D

Latest activity

Jul 2020