Encoded Therapeutics Aims to Push Boundaries of Gene Therapy With $104 Million Financing

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South San Francisco-based Encoded Therapeutics secured $104 million in a Series C financing round that will be used to support development of innovative gene therapy treatments for multiple genetic disorders, including its lead program for Dravet syndrome, a type of epilepsy.

Encoded, which had been incubated by Illumina Accelerator, said its approach to gene therapy will address “key limitations of current gene therapy technology” and will be able to unlock new treatment opportunities. Encoded said its precision gene therapy platform has the potential to enable the development of gene therapies with greater cell-type selectivity, increased potency and the ability to modulate the expression of endogenous genes.

Encoded is focused on four core areas of research: neurocircuitry disorders, liver and metabolic disease, neurodegeneration and cardiovascular disease. Initial programs use clinically-validated adeno-associated viral vectors (AAV) and offer the potential to address disorders outside the reach of current gene therapy technology or significantly improve the benefits of gene therapy in established targets. Encoded’s lead program for Dravet syndrome will target the underlying mechanism of the genetic disorder, which is characterized by uncontrolled seizures, ataxia, significant developmental delays and an increased risk of early mortality due to sudden unexpected death in epilepsy. The majority of Dravet Syndrome cases are caused by loss-of-function mutations in the SCN1A gene. Encoded said the SCN1A gene exceeds the packaging capacity of adeno-associated virus deliveries. Targeting of a specific neuronal cell type, GABAergic inhibitory interneurons is required and Encoded said its platform has enabled the design of an AAV therapeutic that targets the relevant cell type and upregulates endogenous SCN1A expression.

Preclinical data from Encoded’s Dravet syndrome program demonstrated that a single dose of its gene therapy is capable of up-regulating SCN1A expression in GABAergic inhibitory interneurons. When observed for sudden unexpected death in epilepsy over a 10 month period, gene therapy-treated Dravet mice were indistinguishable from wild-type mice, whereas about half of the control-treated Dravet mice died.

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Encoded said its approach to gene therapy could enable new treatment paradigms for people with severe genetic disorders. Encoded uses genomics and computational technologies to identify and optimize DNA sequences in the human genome to control gene expression. This gene regulation platform creates opportunities to advance gene therapies for previously untreatable disorders, the company said.

The Series C financing round was supported by existing shareholders, Venrock, ARCH Venture Partners, Illumina Ventures, Altitude Life Science Ventures and Matrix Capital Management. New investors include Menlo Ventures, RTW Investments, Boxer Capital of Tavistock Group and Alexandria Venture Investments.

Kartik Ramamoorthi, co-founder and chief executive officer of Encoded, said the company’s mission is to “develop and commercialize life-changing therapeutics for severe genetic disorders that are not addressable with existing gene therapy approaches.” Ramamoorthi said the support his company has received from its investors will allow it to develop into a “fully-integrated therapeutics company” and also “establish Encoded as a leading innovator in gene therapy.”

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