EMA approves Clinical Trial Application for retinitis pigmentosa therapy from ViGeneron


VG901 is a gene therapy for CNGA1-associated RP.

A scientist looks at genetic data in a lab. Image credit: ©natali_mis – stock.adobe.com

The gene therapy targets retinitis pigmentosa (RP) for patients who have no treatment options. Image credit: ©natali_mis – stock.adobe.com

On Tuesday, ViGeneron GmbH announced the European Medicines Agency (EMA) issued approval for a Clinical Trial Application (CTA) of its new gene therapy, VG901. In a press release, the company stated VG901 uses an adeno-associated virus (AAV) vector to treat CNGA1-associated retinitis pigmentosa (RP). The proprietary AAV, called vgAAV, delivers the CNGA1 gene via intravitreal injection rather than subretinal administration to avoid retinal damage.1

The company, based in Starnberg, Bayern, Germany, detailed preclinical study results in its statement. Previously, ViGeneron observed that VG901 can supplement the CNGA1 gene in a mouse model of RP, and its safety was confirmed in a GLP safety study with six-month post treatment observation.1 Intravitreal injection and the proprietary vgAAV reportedly make the therapy suitable for various routes of administration.

Next, VG901 will be evaluated for tolerability, efficacy and safety in a Phase Ib dose-escalation trial, the company said. These factors will be evaluated in patients who have developed RP due to biallelic CNGA1 mutations. In paediatric or young adult patients, RP initially presents as nighttime blindness. As the disease progresses, patients experience peripheral visual field loss, tunnel vision, central visual impairment and reduced visual acuity before reaching complete blindness. An estimated 1 in 4,000 people in Europe are affected by RP.1

“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,” Dr Caroline Man Xu, co-founder and CEO of ViGeneron, said. “We are excited to develop this first-in-class gene therapy to provide a potential cure for patients who currently have no treatment options."


  1. ViGeneron announces EMA Approval of Clinical Trial Application for VG901, a gene therapy to treat Retinitis Pigmentosa. News release. ViGeneron GmbH, August 22, 2023.

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