GeneNova

Purpose and goal: ** Denna text är maskinöversatt **

Gene therapy enables a powerful and new way to deliver therapeutic effect, by treating the diseased cell directly at the DNA level. Adeno-associated Viruses (AAVs) have been shown in a number of clinical trials and with recently approved drugs to be both a safe and effective way of gene therapy. Despite this, today it is very expensive and complicated to produce this type of drugs.

GeneNova aims to develop a new platform for bioproduction of this type of drug at improved cost and quality to allow for more patients to get access to these kind of curative drugs. Expected results and effects: ** Denna text är maskinöversatt ** The project will enable a sustainable and scalable production process of AAV-based

drugs and enable more such preclinical research projects to be taken to the next steps faster and reach further in the development process, which in the long run would lead to more patients receiving necessary treatment. Approach and implementation: ** Denna text är maskinöversatt ** GeneNova is a multidisciplinary project, which is carried out in collaboration between leading actors in

different areas of expertise. The environment will deliver a coherent platform with a set of tools, including: viral vectors, cell lines, bioproduction process, formulation and new instrumentation as well as improved and simplified electron microscopy with AI methods for evaluation of AAV quality.