Precision gene therapy company Encoded Therapeutics, Inc. (Encoded) announced it has raised $135 million in an oversubscribed Series D round of funding. And the company also announced that its lead asset ETX101 was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+ Dravet Syndrome.

Dravet syndrome is a rare, severe genetic disorder that occurs in about 1 in 16,000 births worldwide. The disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays, and an increased risk of early mortality due to sudden unexpected death in epilepsy (SUDEP). The majority of Dravet Syndrome cases are caused by loss-of-function mutations in the SCN1A gene. And current treatments reduce seizures but do not address the underlying cause of the disorderSCN1A haploinsufficiency.

GV (formerly Google Ventures) led the Series D round of funding with participation from Matrix Capital Management, ARCH Venture Partners, Illumina Ventures, RTW Investments, Boxer Capital, Nolan Capital, HBM Genomics, Menlo Ventures, Meritech Capital, Farallon Capital Management, SoftBank Vision Fund 21, and several additional unnamed Encodeds discovery engine combines biological and computational approaches for identifying and screening human DNA sequences known as regulatory elements at a high throughput scale. And the resulting multi-dimensional large scale datasets are leveraged to design optimal gene therapy expression cassettes which more precisely control transgene expression.

By recapitulating natural patterns of gene expression, the resulting gene therapy vectors are able to provide maximal therapeutic benefit, minimize off-target expression, and address genetic disorders that were previously Encoded also announced that ETX101 was granted Orphan Disease and Rare Pediatric Disease Designation by FDA. Both programs offer incentives for the development of therapeutics for underserved populations.

The proceeds of the Series D round of funding will be used to conduct clinical trial activities including a natural history study to better understand the progression of SCN1A+ Dravet Syndrome as well as first-in-human trials for ETX101. And the funds will support progression of the companys pipeline of gene therapies being evaluated for additional pediatric CNS disorders.

David Schenkein, M.D., general partner and co-leader of GVs life sciences team, will join the Encoded Board of Directors as an observer in connection with the funding round.

KEY QUOTES:

I am incredibly proud of the progress our organization has made over the past year. We have built an outstanding team, further advanced ETX101 towards the clinic, applied our technology to a series of pipeline programs, and expanded our capabilities to become a fully integrated organization. We are grateful to our investors for supporting our vision to transform patients lives with cell type-selective genetic medicines.

Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D.

Since 2019, our company has attracted some of gene therapys most experienced executive leaders to prepare ETX101 for the clinic and beyond. This well-rounded team has made significant advances in manufacturing, clinical development, and regulatory affairs for ETX101. By coupling these with an innovative discovery engine, Encoded is advancing its preclinical pipeline of gene therapies to patients suffering from devastating pediatric CNS diseases.

Encoded board chairman Sean Nolan.

Encodeds cutting edge platform has the potential to impact a broad range of diseases across different organ systems. Importantly, Encoded has translated this innovation into a one-time, first-in-class precision gene therapy candidate for the treatment of SCN1A+ Dravet Syndrome, a devastating pediatric disease with significant unmet medical need. I am enthusiastic to work with this top-tier team in their mission to treat these underserved patients and advance a portfolio of innovative medicines.

Dr. Schenkein

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Encoded Therapeutics Closes $135 Million In Series D Funding - Pulse 2.0