First patient treated with innovative HSV vector therapy for neurogenic bladder
The biotechnology sector marks a significant advancement in genetic medicine with EG 427’s achievement in treating neurogenic bladder using a novel herpes-based vector approach.
Treatment milestone achieved in neurourology
EG 427, a French biotechnology company specialising in genetic medicines for neurological conditions, has successfully treated the first patient with EG110A, a non-replicative herpes simplex virus type 1 (HSV-1) vector-based therapy. This landmark treatment represents the world’s first application of non-replicative herpes-based vectors in neuro-urology, specifically targeting neurogenic detrusor overactivity (NDO) in patients with spinal cord injury.
The Phase 1b/2a open-label, dose-escalation study commenced at Rancho Research Institute of Rancho Los Amigos National Rehabilitation Center in the greater Los Angeles area, under the supervision of Dr. Evgeniy Kreydin’s team. This clinical investigation will eventually enrol 16 adult participants with persistent urinary incontinence following spinal cord injury who regularly perform clean intermittent catheterisation despite standard of care therapy.
Mechanism of action targets bladder neural pathways
EG110A’s therapeutic approach is mechanistically distinct from current treatment options. The non-replicating HSV-1 vector has been engineered to selectively silence signals from specific bladder sensory neurons that drive bladder muscle overactivity, while preserving motor neuron function and maintaining normal bladder function.
Neurogenic detrusor overactivity represents a significant clinical challenge for patients with spinal cord injury and other neurodegenerative conditions such as multiple sclerosis and Parkinson’s disease. The condition affects approximately 70-84% of patients with spinal cord injury, representing an estimated 300,000-400,000 patients worldwide. Across the seven major pharmaceutical markets, NDO affects approximately 2 million patients and significantly impacts quality of life.
The condition causes uncontrolled urinary incontinence, places patients at risk of kidney damage, and increases susceptibility to urinary tract infections, which can be fatal in 5-10% of the spinal cord injury population. According to the European Association of Urology, incontinence caused by NDO and other indications such as overactive bladder represents a growing economic burden exceeding €69.1 billion in Europe as of 2023.
Clinical study expands across multiple US centres
Philippe Chambon, MD, PhD, Chief Executive Officer at EG 427, described the treatment as “a major milestone in the life of the company” and expressed gratitude to the first patient for participation in the study, calling it “a truly admirable, selfless act.”
The clinical investigation is being conducted at four leading US institutions located across California, Michigan, Pennsylvania and Texas. The study, identified by ClinicalTrials.gov ID NCT06596291, involves a single treatment course consisting of multiple intradetrusor injections of EG110A.
“The treatment of our first patient in our phase 1b/2a study is an important milestone for EG 427. We are looking forward to demonstrating that EG110A has the potential to offer significant improvement over existing therapies in neurogenic detrusor overactivity,” stated Cornelia Haag-Molkenteller, MD, PhD, Chief Medical Officer of EG 427.
Broader implications for genetic medicine in neurology
The advancement comes at a critical time in neurological medicine. A study published in The Lancet Neurology found that neurological diseases affect approximately 3 billion people globally – roughly one in three individuals – with many conditions remaining underserved by current therapeutic approaches.
EG 427’s proprietary HERMES platform technology potentially enables safe, re-dosable and cost-effective delivery of genetic medicines to neural targets. The company’s pipeline aims to address multiple neurological conditions by delivering what they describe as “pinpoint neurotherapeutics” to treat prevalent diseases of the peripheral and central nervous systems.
The company’s vectors can achieve focal transduction in specific regions and selective expression of transgenes in targeted subsets of neurons through sophisticated regulatory elements. The large payload capacity of non-replicative HSV-1 vectors allows for versatile DNA delivery, potentially enabling more sophisticated genetic medicine approaches.
The US Food and Drug Administration cleared the Investigational New Drug application for EG110A in June 2024, positioning EG 427 as a global leader in non-replicating HSV-1 vector technology for neurological applications. If successful, this therapy could represent a significant advancement in the treatment paradigm for neurogenic bladder and related conditions.
