ViGeneron Announces EMA Approval of Clinical Trial Application for VG901, a Gene Therapy to Treat RP
ViGeneron announced that the European Medicines Agency (EMA) has approved the clinical trial application for VG901, a gene therapy to treat CNGA1-associated retinitis pigmentosa (RP). VG901 uses vgAAV, ViGeneron's proprietary adeno-associated 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 1b 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.
“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," Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron, said in a company news release.
In preclinical studies, ViGeneron observed that VG901 has the potential to 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. ViGeneron’s manufacturing partner, WuXi Advanced Therapies (WuXi ATU), has successfully completed GMP manufacturing of the clinical trial material, according to ViGeneron.
VG901 was developed using ViGeneron’s proprietary 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 stated.
"Side by side comparison of in vivo expression 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. Due to 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," the company stated in the news release.