Biotech Industry · global
Ensoma Cuts Staff Again, Pauses Three Preclinical Programs to Focus Resources on EN-374 Gene Therapy
As it seeks new funding, Ensoma is putting its sickle cell disease and oncology research on hold, betting on an in vivo hematopoietic stem cell therapy that has entered a Phase 1/2 trial; the pullback also makes EN-374’s early clinical data critical to whether the platform can continue.
Ensoma is narrowing a gene therapy platform spanning blood disorders and cancer into a test led by a single clinical asset. The biotech company confirmed that it is reducing its workforce for the second time in less than a year and pausing investment and research in programs other than EN-374 to extend its resources and seek additional financing.
The company did not disclose how many employees are affected in this round. The paused preclinical research covers sickle cell disease, immuno-oncology for solid tumors, and hematologic malignancies. Following the restructuring, the top priority is to advance EN-374 for the treatment of X-linked chronic granulomatous disease and thereby obtain clinical validation of Ensoma’s in vivo hematopoietic stem cell engineering platform.
X-linked chronic granulomatous disease is a rare inherited immune disorder caused by defects in the CYBB gene. Patients’ phagocytes have difficulty clearing certain pathogens normally, leaving them prone to recurrent severe infections and inflammation. EN-374 is administered intravenously and uses a helper-dependent adenoviral vector to modify hematopoietic stem cells in the patient’s body so that they express a functional CYBB gene. The concept is intended to eliminate the conventional process of extracting stem cells, processing them outside the body, and reinfusing them.
The currently recruiting Phase 1/2 trial has an open-label, single-dose, dose-escalation design and is expected to enroll 15 participants. It will primarily evaluate safety and potential efficacy and identify a dose for use in subsequent studies. Trial registry information estimates that the primary study will be completed in December 2027, but timelines and enrollment for early-stage trials may still change.
Ensoma previously released preliminary safety data after treating the first participant and described it as the first reported clinical treatment experience conducted in vivo and directly targeting hematopoietic stem cells. However, early safety observations from a single participant cannot demonstrate efficacy and are insufficient to characterize less frequent adverse events. Whether EN-374 can consistently correct a sufficient number of cells and how long the effect can be maintained will require data from more patients and longer follow-up.
The layoffs therefore do more than reduce spending; they also concentrate the company’s technological and financial risks in a single asset. Only if EN-374 produces compelling safety and biological signals will Ensoma have an opportunity to demonstrate again that the platform can be extended to other diseases. If financing or clinical progress is impeded, it will become even more uncertain when the paused research can resume.