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Grant recipient

Magdalena Malm

Mammalian cell factory engineering for improved AAV packaging and release
Grant amount: DKK 8.558.315

Genetic diseases are caused by errors in our genetic material that encodes critical functions in our bodies. To correct such errors has long been a goal for researchers and since 2017, there have been major advancements in the development of so-called gene therapies based on adeno-associated viruses (AAVs). This virus is not causing diseases in humans and can be altered to deliver a correct gene into patients. Such therapies can treat diseases like genetic blindness and hemophilia B. However, a major limitation with these drugs is that they are very expensive to produce, resulting in doses costing up to 3.5 million USD. This is partly due to inefficient production by so called cell factories and low quality of the produced AAVs. This project aims to provide insight into the host environment of the cell factories to pinpoint what components can be altered to improve them. Ultimately, the collected knowledge will be used to generate more sustainable and efficient AAV cell factories.

Magdalena Malm
Magdalena Malm
Senior Researcher, Department of Protein Science, Royal Institute of Technology (Sweden)