The European Medicines Agency has approved the Clinical Trial Application for VG901, a potentially transformative gene therapy to treat CNGA1-associatedretinitis pigmentosa (RP), an ocular disorder currently lacking approved therapies.

Developed by the German based biotech ViGeneron, VG901 uses vgAAV, a proprietary adenoassociated virus (AAV) vector to deliver the CNGA1 gene via intravitreal injection, thereby reducing the risk of retinal damage, which is associated with subretinal administration.

The Phase Ib dose-escalation clinical trial will evaluate safety, tolerability, and efficacy of VG901 in patients with RP due to biallelic CNGA1 mutations. This trial is part of an international regulatory strategy for the clinical development of VG901. Further interactions with regulatory authorities are currently being prepared.

Dr Caroline Man Xu, co-founder and CEO of ViGeneron said, “This is an important step in our mission to bring a novel gene therapy to patients born with CNGA1 mutations, to save their eyesight, potentially. We are excited to develop this first-in-class gene therapy to provide a potential cure for patients who currently have no treatment options.”

In preclinical studies, ViGeneron observed that VG901 has the potential to supplement the CNGA1 gene in a mouse model of RP. A safety study applying a single intravitreal injection followed by a six-month post-treatment observation period confirmed its safety. Furthermore, ViGeneron’s manufacturing partner, WuXi Advanced Therapies, has achieved standards for Good Manufacturing Practices of the clinical trial material, as set out by the United States Food and Drug Administration.