Development of novel therapies for genetic diseases using the Gentrafix platform

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Bitrobius Genetics aims to develop cures for three important and prevalent genetic diseases: cystic fibrosis, muscular dystrophy and myotonia congenita, as well as hereditary breast cancer. We will do this using a new technology called Gentrafix, which will greatly improve the ability of DNA to repair faulty genes using a gene editing technology. It can transport much larger genes than can be carried using current technologies and will be able to reach most of the cells in an affected organ with a single application. For the first time, genetic diseases with no current treatment will be able to be cured, and mutations that lead to or are actively causing cancers can be corrected. The production costs will be lower than those of any competing technology.Genes are made of DNA and represent instructions for making proteins, which in turn form the structure and carry out functions within the body. Genetic diseases and cancers are caused by genes that contain errors, leading to disability in the case of genetic diseases or uncontrolled cell growth in the case of cancers. Gene therapy is a technology that aims to correct these genetic errors. Unfortunately, gene therapy has failed to cure most genetic diseases, and the few therapies that do exist are the most expensive medicines in history. The fundamental problem is that there is no reliable way of getting the correct DNA to every cell in an organ that must be fixed. Most gene therapies use modified viruses which can only infect a few percent of target cells, cannot carry large genes, have a limited ability to spread between cells, can be inactivated by the immune system and must be administered continuously during a patient's life.The project team will construct the disease cures and test them in laboratory experiments, such that at the end of the project, the four primary candidates will be ready to begin the process leading to testing in humans.