Precision gene therapy company Encoded Therapeutics announced late last month that it has successfully closed its Series C financing round, raising $104 million to be used to develop and bring to market therapeutics aimed at several severe genetic disorders. Specifically, the Bay Area-based biotech startup is planning to use the new capital primarily to fund the development of its marquee program: a precision gene therapy intended to treat Dravet syndrome, a rare form of epilepsy.
Encoded first emerged from Illumina’s startup accelerator with seeding from Venrock and ARCH Venture Partners. Participants in the startup’s Series C round include existing investors Illumina, Venrock, ARCH Venture Partners, Matrix Capital Management and Altitude Life Science Ventures; new investors include RTW Investments, Menlo Ventures, Boxer Capital of Tavistock Group and Alexandria Venture Investments.
With the additional capital raised this round, Encoded will be able to work towards human tests for its gene therapy for Dravet syndrome. The goal is simple: the company hopes to be able to demonstrate that its atypical gene therapy approach utilizing DNA sequencing and computing tools can help treat diseases that current gene therapy standards can’t touch. So far, this thesis is only backed by Encoded’s animal tests—but Encoded CEO Kartik Ramamoorthi hopes to begin human studies by early 2021.
In general, Encoded’s entire body of work is founded on a platform aimed at overcoming specific hurdles in the gene therapy space. Traditionally, gene therapy works in one of three ways: by introducing a new healthy copy of a faulty gene, by delivering an entirely new gene or by “knocking out” defective genes. However, Ramamoorthi points out that these three approaches can have issues with cell selectivity, potency and the ability to control endogenous genes.
According to Ramamoorthi, Encoded’s approach is different: “We’ve developed a series of technologies that are both computational and genomics- or sequencing-based that allow us to screen for and identify sequences in vivo that control where and when genes are able to be expressed. We take these sequences and place them in gene therapies—such as adeno-associated viruses (AAVs)—that allow us to control where the adeno-associated viruses are able to express the payload.”
In addition to the precision gene therapy for Dravet syndrome, Encoded plans to advance preclinical programs aimed at combating liver, metabolic and cardiovascular diseases. With the successful close of the Series C funding round, Encoded brought its total funding to $158 million.