Company’s Phase 1/2 Clinical Trial of EN-374 in X-linked Chronic Granulomatous Disease is Now Open for Patient Enrollment

Ensoma, an in vivo hematopoietic stem cell (HSC) engineering company with a mission to advance the future of medicine through one-time therapies, today announced the closing of a $53 million financing from its existing syndicate of top-tier investors. The financing will support Ensoma’s recently initiated Phase 1/2 clinical trial for EN-374 in X-linked chronic granulomatous disease (X-CGD) and support key clinical readouts, while continuing to expand and refine the potential global impact of the company’s in vivo engineered cell therapy platform in immuno-oncology and sickle cell disease.

Investors include Gilead (Nasdaq: GILD), which has also appointed an executive to the Ensoma board of directors as part of its investment, as well as 5AM Ventures, Catalio Capital Management, Cormorant Asset Management, Delos Capital, F-Prime, the Gates Foundation, Hanwha Impact Partners, Mirae Asset Financial Group, Qatar Investment Authority (QIA), RTW, Solasta Ventures, SymBiosis and Viking Global Investors.

“The $53 million financing reflects growing interest in our platform’s potential to address serious diseases through one-time, outpatient treatments,” said Jim Burns, CEO of Ensoma. “This is a defining moment for Ensoma as we are now a clinical-stage company and recruiting participants for our Phase 1/2 trial for EN-374 to treat X-linked CGD. With a strong manufacturing foundation in place, as well, we are in a strong position to begin generating meaningful human data for our first-in-class in vivo HSC-directed therapy.”

About EN-374

EN-374 is a first-in-class in vivo hematopoietic stem cell (HSC)-directed therapy for X-CGD. EN-374 employs virus-like particles (VLPs) to deliver payloads that engineer HSCs with the goal of driving sustained expression of a CYBB transgene in neutrophils, thereby aiming to restore function of the infection-fighting NADPH oxidase enzyme complex critical for immune defense in patients having mutations in their CYBB gene. In preclinical studies in animals, EN-374 demonstrated the restoration of CYBB protein expression and NADPH oxidase activity in circulating neutrophils. Ensoma is currently recruiting participants for a Phase 1/2 open-label, single-ascending-dose study of EN-374 in patients with X-CGD. The trial is designed to evaluate the safety and potential efficacy of EN-374. More information including eligibility criteria is available at www.clinicaltrials.gov (NCT06876363).

About CGD

Chronic granulomatous disease (CGD) is a rare, severe genetic disorder that affects approximately 1 in 100,000-200,000 live births, and the median life expectancy for individuals with the condition is around 45 years. X-linked CGD (X-CGD), the most common form of the condition, comprises 60-70% of cases and is caused by CYBB gene mutations that prevent white blood cells called neutrophils from fighting infection. People with CGD are vulnerable to recurrent, severe bacterial and fungal infections, often leading to chronic and life-threatening dysregulated inflammation and serious complications. Current treatments, including antibiotics, antifungals, interferon gamma and allogeneic stem cell transplantation, offer limited benefit and/or come with significant burdens.

About Ensoma

Ensoma is developing potentially curative medicines for genetic diseases, immune disorders and cancer through in vivo hematopoietic stem cells (HSCs) engineering. Our platform combines proprietary base editing or high-efficiency gene integration systems with high-capacity virus-like particles (VLPs) toward our goal of providing one-time administration of durable genetic medicines. The delivery system is based on VLPs that preferentially bind to HSCs, delivering DNA to the nucleus. With a 35-kilobase cargo capacity, these VLPs can carry a diverse range of sophisticated genomic engineering tools capable of introducing changes from single base edits to large multi-gene insertions, along with control elements for HSC-lineage cell specific expression. Ensoma is supported by top-tier investors and a passionate team committed to a bold, global vision for genetic medicines. Ensoma is based in Boston. For more information, visit ensoma.com.

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