A new $53 million financing round has been successfully secured by Ensoma, an innovative company focused on developing one-time treatments by engineering hematopoietic stem cells (HSCs) directly within the body. This significant capital infusion, provided by a syndicate of top-tier existing investors including Gilead, 5AM Ventures, and the Gates Foundation, will enable Ensoma to achieve key milestones. The primary use of the funds is to support the company's recently initiated Phase 1/2 clinical trial for its lead program, EN-374, which is designed to treat X-linked chronic granulomatous disease (X-CGD). The financing also fuels the continued expansion and refinement of Ensoma's broader in vivo engineered cell therapy pipeline, with promising applications in immuno-oncology and sickle cell disease.

The clinical trial for EN-374 represents a pivotal moment for Ensoma, making it a clinical-stage company. EN-374 is a first-in-class in vivo therapy that uses virus-like particles (VLPs) to deliver a payload directly to HSCs. The goal is to correct the genetic mutation at the root of X-CGD by introducing a functional CYBB gene, thereby enabling the production of a critical enzyme (NADPH oxidase) that is essential for the immune system's ability to fight off infections. This innovative approach aims to provide a durable, one-time treatment option for patients with X-CGD, a severe genetic disorder that leaves them vulnerable to recurrent and life-threatening bacterial and fungal infections. The company's platform is distinct due to the large 35-kilobase cargo capacity of its VLPs, allowing them to carry sophisticated genomic engineering tools capable of making precise genetic changes. This unique delivery system holds great promise for a wide range of genetic diseases and immune disorders, offering a potentially curative, outpatient solution without the burden of traditional cell therapy.