私营基因治疗公司 Encoded Therapeutics 完成 2.75 亿美元 F 轮融资，推进 Dravet 综合征资产 ETX101 关键性研究并自建商业化产能

Neuro-focused Encoded Therapeutics has bagged a $275 million series F round to support its Dravet syndrome asset and scale up its internal manufacturing capabilities.
The proceeds will support a pivotal study of ETX101 in infants and young children with SCN1A-positive Dravet syndrome, a rare form of genetic epilepsy caused by mutations in the SCN1A gene, according to a Wednesday release.
The round was co-led by Google Ventures and another, undisclosed, healthcare fund, with participation from the likes of ARCH Venture Partners, Janus Henderson Investors, RTW Investments and SoftBank Vision Fund.
In addition to the commercial manufacturing build-out, the funding will also support a 2027 IND submission for ETX301, an AAV9-based vectorized microRNA gene therapy for post-amputation neuroma pain.
In May, Encoded dosed its first patient in its phase 2 trial of ETX101, an AAV9-based therapy designed to boost expression of the SCN1A gene in inhibitory neurons. The biotech has been billing ETX101 as the first one-time therapy to address the underlying genetic cause of Dravet syndrome.
The company laid off 29% of its staff in February 2025 to fund a phase 1/2 trial of the drug. That sacrifice appeared to pay off, with the study demonstrating a 78% reduction in seizures at the end of the year.
“With pivotal development underway, this financing gives us the resources to advance ETX101 toward registration while continuing to build the capabilities and pipeline that will define Encoded’s next stage,” CEO Kartik Ramamoorthi, Ph.D., said in the release.
Brendan Bulik-Sullivan, Ph.D., general partner at GV, said the “emerging clinical profile of ETX101 is increasingly compelling, with durable seizure control and promising neurodevelopmental signals reinforcing its potential as a transformative therapy.”
Encoded is not alone in its pursuit of a Dravet syndrome therapy targeting the SCN1A gene. Last year, Biogen boosted its R&D portfolio in the area with a $165 million upfront payment for U.S. rights to Stoke Therapeutics’ zorevunersen, an antisense oligonucleotide designed to boost functional NaV1.1 protein production in brain cells from the non-mutated copy of the SCN1A gene to treat the underlying cause of Dravet syndrome. Stoke and Biogen expect a phase 3 readout in the middle of next year.