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ViGeneron Gets EMA Go-Ahead for Retinitis Pigmentosa Gene Therapy Trial

NEW YORK – ViGeneron on Tuesday said the European Medicines Agency has granted permission to begin a clinical trial of its investigational gene therapy VG901 in patients with CNGA1-associated retinitis pigmentosa.

VG901 uses ViGeneron's proprietary adeno-associated virus vector to deliver a normal CNGA1 gene via intravitreal injection. CNGA1 mutations are known to cause between 2 percent and 8 percent of autosomal recessive retinitis pigmentosa, an inherited, rare eye disorder that starts as nighttime blindness in children and progresses to total blindness later in life.

Munich, Germany-based ViGeneron plans to study the safety, tolerability, and efficacy of VG901 in a Phase Ib dose-escalation trial involving patients who have retinitis pigmentosa due to biallelic CNGA1 mutations. The firm is currently developing an international regulatory strategy for this drug, and this trial is part of that plan.

In preclinical studies, VG901 appeared to be safe and delivered the normal CNGA1 gene in a mouse model of retinitis pigmentosa. The company believes that its proprietary vgAAV-based gene therapy delivered via intravitreal injection will enable easier administration and broader vector distribution compared to subretinal injections. ViGeneron is manufacturing the gene therapy with WuXi Advanced Therapies and said WuXi has successfully completed manufacturing clinical trial materials under good manufacturing practices standards.