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ViGeneron announces EMA approves clinical trial application for VG901 gene therapy to treat retinitis pigmentosa


The company noted that its Phase Ib dose-escalation clinical trial will evaluate safety, tolerability and efficacy of VG901 in patients with RP due to biallelic CNGA1 mutations.

(Image Credit: AdobeStock/Brenda Blossom)

(Image Credit: AdobeStock/Brenda Blossom)

ViGeneron GmbH announced the European Medicines Agency (EMA) has approved the Clinical Trial Application (CTA) for VG901, a potentially transformative gene therapy to treat CNGA1-associated Retinitis Pigmentosa (RP), an ocular disorder currently lacking approved therapies. VG901 uses vgAAV, ViGeneron's proprietary adeno-associated virus (AAV) vector to deliver the CNGA1 gene via intravitreal (IVT) injection, thereby reducing the risk of retinal damage, which is associated with subretinal administration.

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

Caroline Man Xu, PhD, co-founder and CEO of ViGeneron, applauded the news,

“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,” she said in the news release. “We are excited to develop this first-in-class gene therapy to provide a potential cure for patients who currently have no treatment options.”

According to the news release, in its preclinical studies, ViGeneron observed that VG901 potentially could supplement the CNGA1 gene in a mouse model of RP. In addition, a GLP-safety study applying a single intravitreal injection followed by a 6-month post treatment observation period confirmed the safety of VG901.

Moreover, according to the release, ViGeneron’s manufacturing partner, WuXi Advanced Therapies (WuXi ATU), has successfully completed GMP manufacturing of the clinical trial material.

The company noted in its news release it developed VG901 using its next generation gene therapy technology platform vgAAV – a platform based on AAV2 – to be delivered by IVT. This improved vector provides a greater ease of administration and wider vector distribution compared with subretinal injection, commonly used in retinal gene therapy studies.

ViGeneron noted in the news release its side by side comparison of in vivoexpression versus a competitor capsid demonstrated superior IVT transduction efficiency. In addition, IVT delivery avoids the risk of retinal damage associated with subretinal administration and is also less invasive than suprachoroidal injection.

The company added in its news release that as a result of its ability to cross barriers, this vector is suitable for various routes of administration and target organs. It can be combined with our second platform, which allows delivery of genes larger than 5kb, as required for some more prevalent rare diseases.

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