ViGeneron Commences Phase 1b Trial with First Patient Treated Intravitreally for Retinitis Pigmentosa with VG901

ViGeneron GmbH, a clinical-stage gene therapy firm, has initiated a Phase Ib clinical trial for VG901, a novel treatment for retinitis pigmentosa (RP) caused by CNGA1 gene mutations. The therapy, which is the first of its kind, aims to provide the CNGA1 gene to the photoreceptor cells in the retina. 

Utilizing ViGeneron's advanced vgAAV technology, VG901 is delivered intravitreally, which simplifies the procedure, ensures a wider distribution of the vector, and reduces the risk of retinal damage associated with subretinal injections. The FDA has recognized VG901 with Orphan Drug Designation, highlighting its potential to address a significant unmet medical need. The clinical trial, which is open-label and involves a single-arm dose-escalation, is assessing the safety, tolerability, and preliminary efficacy of VG901. 

The study is a crucial step in validating the vgAAV platform, which has shown higher transduction efficiency and allows for less invasive delivery methods. The first patient has already been dosed, marking a significant milestone for the company and the patients it seeks to help. RP is a group of eye disorders leading to progressive vision loss, with symptoms starting as night blindness and potentially progressing to complete blindness. It is the most prevalent inherited retinal disease, affecting an estimated 1 in 3,500 to 1 in 4,000 individuals in the U.S. and Europe. Mutations in the CNGA1 gene account for approximately 2% to 8% of autosomal recessive RP cases. ViGeneron is focused on developing gene therapy solutions for ophthalmic diseases and is also collaborating with biopharmaceutical companies in other therapeutic areas. The company's three innovative gene therapy platforms are designed to overcome the limitations of current AAV-based therapies. 

The vgAAV platform, which is used for VG901, offers superior transduction efficiency and facilitates new administration routes. The REVeRT platform is capable of reconstructing large genes in any tissue, and the AAV Transactivation platform uses CRISPR-Cas technology to regulate gene expression in vivo. ViGeneron, founded in 2017 and based in Munich, Germany, is privately owned and led by a team with extensive experience in AAV vector technology and clinical ophthalmic gene therapy. 

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