Encoded Therapeutics reeled in $104 million in series C cash to bankroll the development of its lead program: a precision gene therapy for Dravet syndrome, a rare form of epilepsy. The Bay Area biotech will also use the funds to push its preclinical programs and come up with new treatments for severe genetic disorders.
The company’s work is based on a platform designed to overcome hurdles it has identified in the gene therapy space. Gene therapies tend to work in one of three ways: They deliver a healthy copy of a faulty gene, introduce a new gene into the body or “knock out” a defective gene. But they can run into problems with cell selectivity, potency and the ability to control endogenous genes, Encoded CEO Karthik Ramamoorthi, Ph.D., told FierceBiotech.
“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,” Ramamoorthi said. “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.”