AVT was founded by three scientific directors, formerly with Genetic Therapy, Inc., a Novartis Company, to commercialize their pivotal research in localized, ocular-based gene therapy. AVT is the only gene-based medicine company that is devoted to eye applications. The founders bring with them the bulk of the technology that they developed at Novartis in a comprehensive and favorable licensing package. Importantly, AVT believes it has overcome the barriers that have encumbered previous gene therapy trials. A critical success factor is in targeting diseases of the eye where there are clear clinical pathways, predictable timelines, excellent pre-clinical models, and a high likelihood of therapeutic success. AVT is targeting large, underserved markets where its products are anticipated to have major competitive advantages.
AVT's initial focus is on the development of a gene-based medicine product for wet age-related macular degeneration. The company's first product, AVT-1, will consist of an AVT vector engineered to deliver Kininostatin, a potent and broadly efficacious anti-angiogenic factor. Following a single intra-ocular administration, the vector will genetically modify a small number of cells in the back of the eye. The modified cells will then secrete Kininostatin, which will diffuse throughout the eye and block the growth of the new blood vessels that cause the disease. Importantly, the delivery of Kininostatin will be continuous and sustained for years. Moreover, Kininostatin blocks defective new blood vessel growth irrespective of the underlying angiogenic stimuli and has a significant advantage over competing products, which target only a single angiogenic stimulus, VEGF. Once having demonstrated efficacy in wet age-related macular degeneration, AVT will move into diabetic proliferative retinopathy with this same product.
The company's second drug candidate, AVT-2, is designed to treat retinitis pigmentosa (RP) and dry age-related macular degeneration The product will consist of the AVT vector engineered to deliver RdCVF, a newly discovered, first-in-class, retinal survival factor that functions to protect and prolong the life of photoreceptors. This product has the potential to open the door to treatments for diseases that have previously been unapproachable.
Finally, AVT is seeking to moderate investor risk by investigating an additional therapeutic gene, T2-TrpRS, for use in wet age-related macular degeneration. T2-TrpRS has potent anti-angiogenic activity in ocular animal models. – less – More from ZoomInfo »