RTW backs Encoded Therapeutics In June, RTW Venture Fund participated in a $135m Series D financing round for Encoded Therapeutics, together with other investment firms. Encoded is a US-based privately held preclinical stage biotech company developing precision gene therapies for a broad range of severe genetic disorders.

This was RTWs seventh investment following its IPO in October 2019 and its second gene therapy company.

Yesterday, Encoded announced thatits lead asset, ETX101, was granted Orphan Drug Designation (ODD) and RarePediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+Dravet Syndrome. Proceeds from the Series D fundraise 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. Additionally, the funds will support progression of the companys pipeline of gene therapies being evaluated for additional pediatric CNS (central nervous system) disorders.

Dravet syndrome is a rare, severe genetic disorder that occurs in approximately 1 in 16,000 births worldwide.The disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays and anincreased risk of early mortality due to sudden unexpected death in epilepsy (SUDEP). The majority of DravetSyndrome cases are caused by loss-of-function mutations in the SCN1A gene. Current treatments reduceseizures but do not address the underlying cause of the disorder SCN1A haploinsufficiency. More informationabout Dravet Syndrome can be found at http://www.dravetfoundation.org.

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RTW backs Encoded Therapeutics - QuotedData