A next generation
gene therapy company
At ViGeneron, we are dedicated to developing innovative gene therapies
to treat ophthalmic diseases with high unmet medical need.
Our two novel gene therapy platforms are designed to overcome the limitations of existing adeno-associated virus (AAV)-based gene therapies and enable the delivery of larger genes as well as superior transduction and less invasive administration routes.
ViGeneron’s pipeline in gene therapy addresses ophthalmic diseases with high unmet medical need, including two lead programs in development for inherited retinal diseases where no approved treatment options are currently available.
The vgAAV vector platform, based on novel engineered AAV capsids, enables a superior transduction of target cells and is designed to efficiently cross biological barriers. These attributes allow vgAAV vectors to target a broad spectrum of cell types in the retina, enabling less invasive intravitreal treatments. Moreover, our vgAAV vectors have potentials to target other tissues, such as the central nervous system.
The REVeRT vector platform overcomes current AAV genome size limitations (>5Kb). This platform uses an innovative vector approach to pack split genes into individual vgAAV vectors and generate a full-length protein via mRNA trans-splicing. It achieves high reconstitution efficiency at the mRNA and protein levels and is broadly applicable in various disease areas.
The novel techniques we develop are based on well-founded scientific results. Together with leading biopharmaceutical partners, we are also addressing other exciting opportunities beyond ophthalmology, potentially in the fields of neuroscience, cardiovascular and liver.