Encoded Therapeutics Secures $104 Million Series C Financing to Advance Gene Therapy for Dravet Syndrome

VCBeat (WeChat ID: vcbeat) learned from foreign media that on June 26, U.S. local time, gene therapy company Encoded Therapeutics announced the completion of a $104 million Series C financing round. The proceeds will be used to advance its R&D pipeline for Dravet syndrome and to leverage its platform to develop new therapies capable of treating severe genetic diseases.

It is reported that this round of financing was jointly participated in by existing investors Venrock, ARCH Venture Partners, Matrix Capital Management, Illumina Ventures, and Altitude Life Science Ventures, as well as new investors Menlo Ventures, RTW Investments, Boxer Capital, and Alexandria Venture Investments.

Encoded Therapeutics is a biotechnology company headquartered in San Francisco, USA, that develops precision gene therapies for severe genetic diseases. The company is committed to overcoming the bottlenecks of viral gene therapy and unlocking the potential of genomics-driven precision medicine. Encoded Therapeutics possesses a precision gene therapy technology platform capable of developing a variety of gene therapies with enhanced cell selectivity and improved regulation of endogenous gene expression. By addressing key limitations of current gene therapy technologies, Encoded Therapeutics has created new therapeutic opportunities.

Currently, Encoded Therapeutics is primarily focused on Dravet syndrome, a severe and rare genetic disorder. Approximately 16,000 infants with Dravet syndrome are born worldwide each year. The condition is characterized by uncontrolled seizures, ataxia, and developmental delays. Most cases of Dravet syndrome are caused by mutations in the SCN1A gene.

Gene therapy can theoretically treat most genetic disorders for which underlying pathogenic mechanisms have been identified. However, Dravet syndrome is widely recognized as a particularly challenging area for gene therapy because the SCN1A gene exceeds the packaging capacity of adeno-associated virus (AAV) vectors. Effective modulation of this gene requires targeting specific neuronal populations and suppressing SCN1A expression in inhibitory interneurons via GABAergic mechanisms. Encoded Therapeutics’ technology platform enables the design of AAV-based therapeutics tailored to relevant cell types, upregulating endogenous SCN1A expression to treat Dravet syndrome.

“Our mission is to develop gene therapies for severe genetic disorders that currently lack standard treatments,” said Dr. Kartik Ramamoorthi, co-founder of Encoded Therapeutics. “The funding we have secured from investors will help Encoded Therapeutics evolve into a fully integrated therapeutic company and establish a leadership position within the industry.”

“By addressing some of the current limitations of gene therapy, Encoded Therapeutics is poised to become a leader in the field. We are excited to support Encoded Therapeutics in bringing novel gene therapies to diseases that currently lack treatment options,” said Dr. Bryan Roberts, Partner at Venrock.

(Compiled by Wang Chan)