The activity of AAVantgarde Bio is focused on the development of new in vivo gene therapy platforms for hereditary forms of blindness capable of overcoming one of the main current limitations, the "capacity" of the vectors: the systems already in use have in fact a capacity to limited gene transport. AAVantgarde Bio will try to respond to this challenge thanks to a technology developed for the first time in the Tigem laboratories by the research group led by Alberto Auricchio, head of the institute's Molecular Therapies program and full professor of Medical Genetics at the University "Federico II" of Naples. In particular, AAVantgarde Bio owns two different platforms that allow for the transfer of large "fragmented" genes, while ensuring that once inside the cell, the complete therapeutic protein is then produced correctly. The company is validating these platforms in two genetic disorders: retinitis pigmentosa associated with Usher syndrome type 1B and Stargardt syndrome. The new funding will be used to complete two "proof of concept" studies on volunteers with these two pathologies, preparatory to the start of the clinical phase. It is also the company's intention to explore the possibility of applying these technologies in other areas as well, not just for eye diseases.
«Finally these two platforms we've been working on for years, which have proven to work well in the laboratory, will be able to be tested in view of a future application in humans, with the hope of bringing benefits to people with vision diseases to date incurable". For the general manager of Telethon Francesca Pasinelli «the substantial funding obtained from AAVantgarde Bio confirms the value of the technological platform developed by the research group led by Alberto Auricchio. This is a fine example of synergy between academic and industrial research: an excellent programme, suitably enhanced in terms of development and technology transfer potential, has been intercepted by industry and we hope it will be able to generate products useful to the community.“It is a great satisfaction to begin to see the fallout of this collaboration for our main 'shareholders', who are people with rare genetic diseases”.