GW Pharmas Epidiolex made history in 2018 when the cannabis-based treatment scored an FDA nod for Dravet syndrome, a genetic disorder marked by intractable seizures. But children with Dravet face much more than just seizures. Enter Encoded Therapeutics, which just raised $135 million to advance a precision gene therapy that could treat the disease in its totality.

The series D financing will bankroll a natural history study, slated for the second half of 2020, of Dravet syndrome stemming from mutations in the SCN1A gene, as well as a phase 1 study to start in 2021. Drawn from a long list of marquee investors including GV, Matrix Capital Management and Arch Venture Partners, the capital will also support a clutch of earlier-stage gene therapies for other pediatric central nervous system (CNS) disorders.

Encoded aims to surmount hurdles in the gene therapy space, including the treatments ability to zero in on specific cells, their potency and their ability to control endogenous genes, CEO Kartik Ramamoorthi, Ph.D., told Fierce Biotech in a previous interview.

RELATED: Encoded Therapeutics bags $104M to propel 'precision gene therapy' for Dravet syndrome

Rather than "knock out" defective genes, introduce a new gene into the body or replace a faulty gene with a healthy copy, Encoded targets pieces of DNA that control gene expression. Its technology screens for and identifies sequences in the human genome dubbed regulatory elements because they control the expression of other genes.

Theyre fundamentally important as to why different types of cells are unique and operate differently, Ramamoorthi said. We can incorporate these sequences into any gene delivery vehicle of interest; were focused on adeno-associated viruses as an initial application.

Its lead program, ETX101, is designed to remedy mutations in the SCN1A genewhich causes the majority of Dravet casesby boosting expression of SCN1A in a specific cell type in the brain. This way, Encoded aims to tackle gait problems, impaired coordination and developmental delays.

Encoded hasnt disclosed its programs beyond ETX101, but Ramamoorthi said they target diseases that are similar to Dravet syndrome.

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, he said.

RELATED: Taysha Gene Therapies hits the ground running with $30M, 15 programs

The latest financing comes about a year after Encodeds $104 million series C round. Since then, the company has filled out its team with gene therapy trailblazers like Chief Manufacturing OfficerAndrew Stoberand Chief Regulatory OfficerNancy Boman, M.D., Ph.D., both of whom were on the AveXis team that developed and submitted the gene therapy Zolgensma for FDA approval.

Theyve done it before in gene therapy and theyre hungry for more, Ramamoorthi said.

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Encoded Therapeutics bags $135M to push 'precision gene therapy' into the clinic - FierceBiotech