Vania Broccoli, one of the founders of Repron Therapeutics, has been involved for years in research on Rett syndrome, a very serious neurodevelopmental disorder that represents the second cause of cognitive disabilities after Down syndrome.
In this capacity his studies on gene therapy have received pre seed funding from CDP-Venture Capital as part of the Extend program, National Technology Transfer Hub entirely dedicated to the biopharmaceutical sectorreceived pre seed funding from CDP-Venture Capital as part of the Extend program, National Technology Transfer Hub entirely dedicated to the biopharmaceutical sector.
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Broccoli was among the guests of the Italian Tech Week which was held at the Officine Grandi Riparazioni in Turin.
āExtendās contribution is not only financial but strategic as it is the only program on the Italian market that contributes to generating a dedicated scientific development plan, up to a maximum of 18 months, to reach an inflection point whose data , validated in the industrial sector, will be fundamental to establish the spin-offā.
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With Rett syndrome we are talking about āa group of neuroinfantile diseases, which are considered rare and without a specific cure and which together represent the major cause of cognitive disabilities, severe motor disorders, autism in children. In most cases they are of genetic origin. In the last 20 years, pharmacological treatments have not given satisfactory results. A solution may come from gene therapy. Broccoli is one of the researchers who has worked on a strategy for the āreintroduction of the healthy geneā into laboratory models of this disease thanks also to the ongoing support of Telethon. A path that also promises recovery of the damage caused. āWe have incontrovertible data on this ā he explains ā on our animal modelsā.
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