Retinitis pigmentosa is one of the most common forms of hereditary retinal degeneration. The disease typically begins with night blindness, followed by vision loss and a gradual restriction of the visual field. It usually ends in blindness. Retinitis pigmentosa occurs in approximately 1 in 4,000 Europeans. Researchers have now identified over 80 different genes that trigger this previously incurable disease. With its active substance VG901, Vigeneron focuses on a mutation of the CNGA1 gene.
In preclinical studies in mice, Vigeneron has already demonstrated the efficacy of the substance. Subsequently, the safety of VG901 was investigated. Now, the startup is beginning phase I of clinical trial – following approval by the European Medicines Agency. In this phase, with the help of voluntary test subjects, it will be examined whether the medicine raises no relevant safety concerns and reaches the target area in the body and remains there long enough to exert its effect. Additionally, in this phase, the new drug should provide preliminary proof of its therapeutic value.
Vigeneron relies on its own developed gene therapy platform
The active substance VG901 is based on the gene therapy platform vgAAV, which Vigeneron has developed itself. This is the result of directed in vivo evolution of adeno-associated viruses (AAV). These are popular in gene therapy for delivering genetic material into target cells (viral vector). AAV appear particularly suitable because they are not associated with diseases and the virus’s own genetic material rarely integrates non-specifically into the host cell’s genome.
Thanks to these properties, vgAAV vectors can target a broad spectrum of cell types in the retina. The special feature is that the active substance can be administered intravitreally. This means it is delivered into the vitreous of the eye using a syringe. Other potential drugs for combating retinitis pigmentosa, by contrast, must be administered subretinally, meaning directly beneath the retina. However, vgAAV is also generally applicable to uses outside ophthalmology, according to Vigeneron.
Dr. Caroline Man Xu, co-founder and CEO of Vigeneron, says:
“This is an important step in our mission to save the eyesight of patients born with CNGA1 mutations using novel gene therapy. We look forward to developing this first gene therapy of its kind to enable a cure for patients for whom there are currently no treatment options.”
Vigeneron was founded in 2017 as a spin-off of Ludwig Maximilians University (LMU) Munich by Caroline Man Xu. Dr. Martin Biel, professor of pharmacy and chair of the Department of Pharmacy at LMU, and Dr. Stylianos Michalakis, professor of ocular gene therapy at LMU, serve as scientific co-founders. The startup’s investors include pharmaceutical company WuXi Apptec from China and Sequoia Capital China. Vigeneron is currently based at the IZB in Planegg.
