Ensoma's EN-374 Gains Rare Pediatric and Orphan Drug Status for Chronic Granulomatous Disease

Ensoma, a biotech company focused on genomic medicines, has announced a significant milestone for its lead program, EN-374, which targets X-linked chronic granulomatous disease (X-CGD). This rare genetic disorder affects the immune system, making patients susceptible to severe and recurring bacterial and fungal infections. The U.S. Food and Drug Administration (FDA) has granted EN-374 rare pediatric disease and orphan drug designations, recognizing the pressing need for more effective treatment options.

Chronic granulomatous disease (CGD) is a severe condition that hinders the immune system's ability to combat infections, often resulting in chronic inflammation and serious health complications. Affecting approximately 1 in 100,000 to 200,000 live births, the median life expectancy for individuals with CGD is about 45 years. The most common form of this disease, X-CGD, comprises 60-70% of all CGD cases and is caused by mutations in the CYBB gene. These mutations impair the function of neutrophils, a type of white blood cell, in fighting infections.

EN-374 is designed to address X-CGD by delivering a functional CYBB gene directly to hematopoietic stem cells (HSCs) in patients. The therapy aims to restore the ability of neutrophils to fight infections by promoting the expression of the CYBB gene specifically in these cells. This innovative approach represents the first in vivo HSC-directed treatment for X-CGD, building on mechanisms that have already shown success ex vivo.

Current treatment options for X-CGD, such as antibiotics, antifungals, interferon gamma, and allogeneic stem cell transplantation, are either limited in effectiveness or come with significant challenges. EN-374 offers a promising alternative by potentially improving upon the benefits of ex vivo gene therapies, in a manner that is simpler, safer, and more accessible to patients. Ensoma plans to submit an investigational new drug application for EN-374 in the first half of 2025.

The rare pediatric disease designation from the FDA is awarded to encourage the development of treatments for serious or life-threatening diseases that primarily affect children and impact fewer than 200,000 individuals in the U.S. If a Biologics License Application for EN-374 is approved, Ensoma may qualify for a Priority Review Voucher from the FDA, which can expedite the review process for a future marketing application.

Additionally, the orphan drug designation provides similar encouragement for the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S. This designation offers Ensoma various incentives, including up to seven years of market exclusivity upon approval, tax credits for qualified clinical trials, and exemption from some FDA fees.

Ensoma is at the forefront of developing curative medicines through precision in vivo cellular engineering. Their platform integrates advanced base editing, efficient gene integration systems, and high-capacity virus-like particles to deliver durable genetic therapies as a one-time outpatient procedure. By focusing on in vivo engineering of hematopoietic stem cells, Ensoma aims to address genetic diseases, immune disorders, and cancer effectively. Based in Boston, the company is supported by leading investors and a dedicated team committed to advancing genomic medicine on a global scale.