A next generation
gene therapy company
At VeonGen, we are dedicated to developing innovative gene therapies
to treat ophthalmic diseases with high unmet medical need.
Our three novel gene therapy platforms are designed to overcome the limitations of existing adeno-associated virus (AAV)-based gene therapies to
- achieve superior delivery through less invasive administration routes
- enable the delivery of large genes
- modulate the expression of several genes
VeonGen’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.
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VeonGen’s unique and
proprietary technology platforms
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, heart, pancreas or skeletal muscle.
Our capsids can be inserted into any serotype to change tropism.
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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.
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Transactivation enables modulating the expression of multiple genes at once, independent of gene size. Expression of one or more genes can be amplified, while another gene can be knocked out simultaneously. Our innovative REVeRT platform is a prerequisite for this new, highly versatile approach.
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The novel techniques we develop are based on well-founded scientific results. Together with leading biopharmaceutical partners we are exploring other exciting opportunities beyond ophthalmology in the fields of neuroscience, diabetes, cardiovascular and muscular diseases