Encoded Therapeutics Uses Precision Gene Therapies To Curb CNS Disorders

San Francisco-based Encoded Therapeutics develops precision medicine therapies to address the root of diseases, rather than simply mitigating the symptoms. While other pharma companies work on knocking out unwanted, defective genes, Encoded Therapeutics adds a new gene to the body, or replaces genes that are performing sub-optimally with a fully functioning facsimile.

The company recently raised $135 million in an oversubscribed Series D round of funding led by GV (formerly Google Ventures). Participation also came from Matrix Capital Management, ARCH Venture Partners, Illumina Ventures, RTW Investments, Boxer Capital, and many others. The capital will be used to drive a clinical trial, including a natural history study, on Dravet Syndrome, a severe form of epilepsy occurring in 1 in 16,000 births worldwide.

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The syndrome stems from mutations in the SCN1A gene, which Encoded hopes to further elucidate with a Phase 1 study slated to begin in 2021. The first human trials of their drug ETX101 will be made possible by the funding round. ETX101 was granted Orphan Drug Designation and Pediatric Disease Designation by the U.S. Food and Drug Administration for use as a therapy for Dravet Syndrome. The loss-of-function mutations to the SCN1A gene can lead to uncontrollable seizures, ataxia, severe developmental delays, and even 15-20% mortality rate before reaching adulthood. ETX101 is designed to overcome the mobility and coordination issues and developmental delays that plague those affected by Dravet.

Preclinical studies have shown promise thus far – a single dose dramatically reduces seizures and extends survival in existing mouse models. The Series D round will finally allow Encoded to move the mouse studies over to human trials, and also support the company’s full pipeline of gene therapies in the works to alleviate other pediatric CNS disorders.

“Many of these pediatric disorders result in cell type-selective dysfunction, meaning a particular type of cell in the central nervous system is not operating appropriately, usually due to a single gene dysfunction,” said Kartik Ramamoorthi, Encoded Therapeutics’ CEO. With treatments that hone in on specific cells and their influence over a gene, Encoded hopes to develop solutions that will treat diseases in their totality, rather than just providing a temporary reprieve.