In vivo HSC-directed therapy advancing to IND filing in H1 2025 for treatment of X-linked chronic granulomatous disease (X-CGD)
BOSTON--(BUSINESS WIRE)--Ensoma, a genomic medicines company advancing the future of medicine through one-time, in vivo treatments capable of precisely and durably engineering the hematopoietic system, today announced the U.S. Food and Drug Administration (FDA) has granted both rare pediatric disease and orphan drug designations to the company’s lead program, EN-374, for the treatment of X-linked chronic granulomatous disease (X-CGD). Ensoma anticipates it will submit an investigational new drug application (IND) for EN-374 in the first half of 2025.
Chronic granulomatous disease (CGD) is a rare and severe genetic disorder, which significantly impairs the immune system. It leaves patients vulnerable to recurrent and severe bacterial and fungal infections, often leading to chronic and life-threatening dysregulated inflammation and serious complications. CGD 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-CGD is the most common form of the condition, affecting 60-70% of CGD patients, and is caused by mutations in the CYBB gene that prevent white blood cells called neutrophils from fighting infection. EN-374 is designed to treat X-CGD through the delivery of a functional CYBB gene directly to hematopoietic stem cell (HSCs) in vivo. It uses a promoter for selective expression in neutrophils, the primary cell type affected by X-CGD. Successful expression will restore patients’ ability to fight infection.
“These designations from the FDA highlight the significant medical need in chronic granulomatous disease,” said Drew Dietz, M.D., MSCR, vice president of clinical development at Ensoma. “Current treatments, including antibiotics, antifungals, interferon gamma and allogeneic stem cell transplantation, offer limited benefit and/or come with significant burdens. EN-374 represents the first in vivo HSC-directed therapy for X-CGD, building on a mechanism that has been validated ex vivo. Designed to function for any CYBB mutation, this approach offers the potential to improve upon transformative benefits of ex vivo gene therapies in a way that’s simpler, potentially safer and more accessible for patients. Our team is incredibly proud to be at the forefront of advancing this groundbreaking therapy. We remain on track to submit an IND in the first half of this year.”
Rare pediatric disease designation is granted by the FDA to incentivize development of treatments for serious or life-threatening rare diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the U.S. If a Biologics License Application for EN-374 is approved in the U.S., Ensoma may be eligible to receive a Priority Review Voucher from the FDA.
Orphan drug designation is granted by the FDA to products that prevent, diagnose or treat a rare disease or condition affecting fewer than 200,000 people in the U.S. The designation affords Ensoma the potential for certain benefits, including up to seven years market exclusivity after approval, tax credits for qualified clinical trials and exemption from certain FDA fees.
About Ensoma
Ensoma is developing curative medicines through precision in vivo cellular engineering. Our platform combines class-leading proprietary base editing and high-efficiency gene integration systems with high-capacity virus-like particles (VLPs) to provide one-time and durable genetic medicines in an outpatient procedure. We are focused on in vivo engineering of hematopoietic stem cells (HSCs) to address genetic diseases, immune disorders and cancer. Ensoma is supported by top-tier investors and a passionate team committed to a bold, global vision for genomic medicines. Ensoma is based in Boston. For more information, visit ensoma.com.
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Josie Butler, 1AB
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