Pharma Focus Europe

FDA Grants IND Approval for First Gene Therapy Targeting Common Neurological Conditions – EG 427 Commences Initial Human Clinical Trial

Monday, June 24, 2024

EG 427, a leading biotechnology company, has received FDA clearance for clinical trials of EG110A, a gene therapy targeting Neurogenic Bladder (NDO) in spinal cord injury (SCI) patients. This therapy utilizes a non-replicating vector derived from HSV-1 to selectively inhibit overactive bladder signals from sensory neurons while preserving normal bladder function.

NDO is prevalent among SCI patients and individuals with neurodegenerative diseases, leading to severe urinary problems such as incontinence and posing significant health risks. EG 427 is conducting a phase 1b/2a study at prominent American institutions to address this critical medical need.

Dr. Cornelia Haag-Molkenteller, EG 427's medical director, highlighted the therapy's potential based on promising preclinical results, suggesting it could surpass current treatments with lasting effectiveness and minimal impact on bladder function.

Philippe Chambon, MD, PhD, CEO of EG 427, described the FDA clearance as a milestone, emphasizing their HSV-1 vector platform's potential to offer safe, cost-effective gene therapies for neglected neuro-urological conditions.

EG 427 aims to leverage its unique vector technology to develop targeted neurotherapies that address significant global medical needs in neurological diseases.



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